RNA Processing I: Splicing notes

Eukaryotic Split Genes and Splicing

  • Eukaryotic genes are interrupted by noncoding DNA, unlike bacterial genes.

  • RNA polymerases transcribe both coding and noncoding regions.

  • The cell removes noncoding RNA from the primary transcript via splicing.

  • Eukaryotes add a 5’ cap and a 3’ poly-A tail to the transcript.

  • All mRNA processing events occur in the nucleus before export to the cytoplasm.

Genes in Pieces

  • Introns: Noncoding, intervening sequences within a gene.

  • Exons: Coding regions of a gene.

  • mRNA genes can have anywhere from 0 to 362 introns, while tRNA genes have 0 or 1.

RNA Splicing

  • Introns are transcribed along with exons in the primary transcript.

  • Splicing removes introns and joins exons to form mature RNA.

    • Possibility 1: Introns are never transcribed

      • Polymerase somehow jumps from one exon to another

    • Possibility 2: Introns are transcribed

      • Primary transcript result, an overlarge gene product is cut down by removing introns

      • This is correct process

Splicing Signals

  • Splicing must be precise.

  • Splicing signals in nuclear mRNA precursors are uniform.

    • The first two bases of introns are GU, and the last two are AG.

    • 5’- and 3’-splice sites have consensus sequences with a branchpoint.

    • Yeast: 5’-AG/GUAAGU-intron-YNCURAC-YnNYAG/G-3’

    • Whole consensus sequences are crucial for proper splicing.

    • Mutations in consensus sequences can lead to abnormal splicing.

Mechanism of Splicing of Nuclear mRNA Precursors

  • Splicing involves a branched intermediate called a lariat.

  • Two-step model:

    • The 2’-OH group of A in the middle of the intron attacks the phosphodiester bond between the first exon and the G at the beginning of the intron, forming the lariat.

      • ApGUAAGUApGUAAGU

    • The 3’-OH left at the end of the first exon attacks the phosphodiester bond linking the intron G to the second exon, forming the exon-exon phosphodiester bond.

      • 5AGG35’-AGG-3’

    • This releases the intron in lariat form.

Signal at the Branch

  • Along with consensus sequences at 5’- and 3’-ends of nuclear introns, branchpoint consensus sequences also occur.

  • Yeast sequence invariant: UACUAAC (e.g. yeast actin gene study).

  • Higher eukaryote consensus sequence is more variable (U47NC63U53R72A91C47)

  • Branched nucleotide is final A in the sequence.

Spliceosomes

  • Splicing occurs on a particle called a spliceosome.

  • Yeast and mammalian spliceosomes have sedimentation coefficients of 40S and 60S, respectively.

  • Spliceosomes contain pre-mRNA, snRNPs, and protein splicing factors.

  • These components recognize key splicing signals and orchestrate the splicing process

snRNPs

  • Small nuclear RNAs coupled to proteins are abbreviated as snRNPs (snurps), small nuclear ribonuclear proteins.

  • The snRNAs can be resolved on a gel:

    • U1, U2, U4, U5, U6

    • All 5 snurps join the spliceosome to play crucial roles in splicing

U1 snRNP

  • U1 snRNA sequence is complementary to both 5’- and 3’-splice site consensus sequences.

    • U1 snRNA base-pairs with these splice sites

    • Brings the sites together for splicing is too simple an explanation

  • Splicing involves a branch within the intron.

  • Genetic experiments have shown that base pairing between U1 snRNA and 5’-splice site of mRNA precursor is necessary but not sufficient for binding. Alternative 5’-splice sites of E1A

U6 snRNP

  • U6 snRNP associates with the 5’-end of the intron by base pairing through the U6 RNA.

  • U6 cross-links to intron at +5, suggesting that the invariant sequence ACA in U6 base-pairs with the conserved UGU (+4 to +6) of the intron.

  • Occurs first prior to formation of lariat intermediate but after first step in splicing.

  • The association between U6 and splicing substrate is essential for the splicing process.

  • U6 also associates with U2 during splicing (Active site).

U2 snRNP

  • U2 snRNA base-pairs with the conserved sequence at the splicing branchpoint.

  • This base pairing is essential for splicing.

  • U2 (nuc 23 and 26-28) also forms base pairs with U6 (nuc 56-59):

    • This region is called helix I

    • Helps orient snRNPs for splicing

  • 5’-end of U2 interacts with 3’-end of U6:

    • This interaction forms a region called helix II

    • This region is important in splicing in mammalian cells, not in yeast cells

U5 snRNP

  • U5 snRNA shows no sequence complementarity but does associate with the last nucleotide in one exon and the first nucleotide of the next exon.

  • This should result in the two exons lining up for splicing.

U4 snRNP

  • U4 base-pairs with U6 through stems I and II and does not play a role in splicing.

  • Its role seems to be to bind and sequester U6.

  • When U6 is needed in a splicing reaction, U4 is removed to allow interaction with U2.

  • Interaction of U6 with U2 involves some U6 bases involved in U4 stem I formation.

snRNP Involvement in mRNA Splicing

  • Spliceosomal complex contains (Substrate, U2, U5 & U6).

  • The complex ready for the 2nd step in splicing can be drawn as a group II intron (self-splicing intron) at the same stage of splicing.

  • Spliceosomal snRNPs substitute for elements at the center of catalytic activity of group II introns at the same stage of splicing.

Spliceosome Catalytic Activity

  • Catalytic center of spliceosome appears to include Mg2+Mg^{2+} and a base-paired complex of 3 RNAs:

    • U2 snRNA

    • U6 snRNA (binds Mg2+Mg^{2+} plays a role in catalysis)

    • Branchpoint region of the intron

  • Protein-free fragments of these RNAs can catalyze a reaction related to the first step in splicing.

Spliceosome Assembly and Function

  • Spliceosome is composed of many components – proteins and RNA.

  • These components assemble stepwise.

  • The spliceosome cycle includes assembly, splicing activity, and disassembly.

  • By controlling assembly of the spliceosome, a cell can regulate quality and quantity of splicing and so regulate gene expression.

Spliceosome Cycle

  • Assembly begins with binding of U1 to splicing substrate forming a commitment complex (CC), a unit committed to splicing out the intron.

  • U2 joins, with help from ATP, to form the A complex.

  • Next, U4-U6 and U5 join to form the B1 complex.

  • U6 dissociates from U4, and displaces U1 at the 5’-splice site:

    • This step is ATP-dependent

    • Activates the spliceosome

    • Allows U1 and U4 to be released

    • Allows U6 to base-pair with U2

  • Activated spliceosome is called the B2 complex.

  • ATP provides the energy to allow lariat formation in the C1 complex.

  • Once another ATP is consumed, the second splicing step occurs resulting in the C2 complex.

  • Mature RNA exits the complex, leaving the intron bound to the I complex.

A Minor Spliceosome

  • A minor class of introns with variant but highly conserved 5’-splice sites and branchpoints can be spliced with the help of a variant class of snRNAs.

  • Cells can contain minor spliceosome with minor snRNAs:

    • U11 performs like U1

    • U12 acts like U2

    • U4atac and U6atac perform like U4 and U6 respectively

    • Use the same U5 as the major spliceosome

Commitment, Splice Site Selection and Alternative Splicing

  • snRNPs do not have enough specificity and affinity to bind exclusively and tightly at exon-intron boundaries.

  • Additional splicing factors are needed to help snRNPs bind.

  • Some splicing factors are needed to bridge across introns and exons and so define these RNA elements.

Exon and Intron Definition

  • The spliceosome can recognize either exons or introns in the splicing commitment process, presumably by assembling splicing factors to bridge across exons or introns.

  • If exons are recognized it is exon definition (higher eukaryotes).

  • If introns are recognized it is intron definition (yeast).

  • Splicing in a given organism typically uses either exon definition or intron definition.

Commitment

  • Commitment to splice at a given site is determined by an RNA-binding protein.

  • This protein binds to splicing substrate and recruits other spliceosomal components.

  • The first component to follow is U1.

  • SR proteins SC35 and SF2/ASF commit splicing on human β-globin pre-mRNA and HIV tat pre-mRNA, respectively.

  • Part of the commitment involves attraction of U1 in some cases.

  • Commitment with different pre-mRNAs requires different splicing factors.

Bridging Proteins and Commitment

  • Because U1 is the first to bind, interacting proteins were sought out (synthetic lethality in U1  MUD2; in MUD2  MSL-5 [BBP]).

  • Yeast commitment complex has a branchpoint bridging protein (BBP) binds to:

    • U1 snRNP protein at the 5’-end of the intron

    • Mud2p near the 3’-end of the intron

    • RNA near the 3’-end of the intron

  • Bridges the intron and could play a role defining intron prior to splicing.

  • Mammalian BBP is SF1 and Mud2p is U2AF65 and may serve the same bridging function to define exons.

3’-Splice Site Selection

  • Splicing factor Slu7 is required for correct 3’- splicing site selection (proper AG at the 3’ of intron).

  • Without Slu7, splicing to correct 3’-splice site AG is suppressed and splicing to aberrant AG is activated.

  • U2AF is also required for 3’- splice site recognition.

  • 65KDa U2AF subunit binds to polypyrimidine tract upstream of 3’-splice site and 35KDa subunit binds to the 3’-splice site AG.

Role of the RNA Polymerase II CTD

  • C-terminal domain of the Rpb1 subunit of RNA polymerase II stimulates splicing of substrates that use exon definition.

  • This does not apply to those that use intron definition to prepare for splicing.

  • CTD binds to splicing factors and could assemble the factors at the end of exons to set them off for splicing.

Alternative Splicing

  • Transcripts of many eukaryotic genes are subject to alternative splicing.

    • This splicing can have profound effects on the protein products of a gene

    • Can make a difference between:

      • Secreted or membrane-bound protein (µs and µm)

      • Activity and inactivity

    • Products of 3 genes in sex determination pathway of the fruit fly are subject to alternative splicing.

  • Female-specific splicing of tra transcript gives:

    • An active product that causes female-specific splicing of dsx pre-mRNA

    • This produces a female fruit fly

  • Male-specific splicing of tra transcript gives:

    • An inactive product that allows male-specific splicing of dsx pre-mRNA

    • This produces a male fruit fly

  • Alternative splicing of the same pre-mRNA gives rise to very different products

    • Alternative splicing patterns occur in over half of human genes

    • Many genes have more than 2 splicing patterns, some have thousands

    • We have 26=642^6 = 64 possible isoforms

Control of Splicing

  • What stimulates recognition of signals under only some circumstances?

  • Exons can contain different sequences

    • Exonic splicing enhancers (ESEs) tend to interact with SR proteins and stimulate splicing (e.g. dsx exon 4 weak 3’ SS for U2AF)

    • Exonic splicing silencers (ESSs) interact with hnRNP proteins and inhibit splicing (e.g. hnRNP A1)

Alternative Splicing Summary

  • Alternative splicing is very common in higher eukaryotes.

  • It represents a way to get more than one protein product out of the same gene and a way to control gene expression in cells.

  • Such control is exerted by splicing factors that bind to splice sites and a branchpoint, and also by proteins that interact with ESEs, ESSs and intronic splicing elements.

Self-Splicing RNAs

  • Some RNAs could splice themselves without aid from a spliceosome or any other protein.

  • Tetrahymena 26S rRNA gene has an intron, splices itself in vitro.

    • Group I introns are a group of self-splicing RNAs

    • Another group, Group II introns also have some self-splicing members

Group I Introns

  • Group I introns can be removed in vitro with no help from protein.

  • Reaction begins with attack by a guanine nucleotide on the 5’-splice site.

    • Adds G to the 5’-end of the intron

    • Releases the first exon

  • Second step, first exon attacks the 3’-splice site.

    • Ligates 2 exons together

    • Releases the linear intron

  • Intron cyclizes twice, losing nucleotides each time (15 + 4 = 19 nt), then linearizes a last time.

Group II Introns

  • RNAs containing group II introns self-splice by a pathway using an A-branched lariat intermediate, like spliceosome lariats.

  • Secondary structures of the splicing complexes involving spliceosomal systems and group II introns are very similar, suggesting a common evolutionary origin.

  • Nuclear pre-mRNA introns may have descended from bacterial group II introns.

  • Group II introns have been found in archaea, cyanobacteria and purple bacteria

Eukaryotic Split Genes and Splicing

  • Eukaryotic genes are interrupted by noncoding DNA, a feature absent in bacterial genes, which primarily contain continuous coding sequences.

  • RNA polymerases transcribe both coding and noncoding regions, necessitating post-transcriptional processing in eukaryotes.

  • The cell removes noncoding RNA from the primary transcript via splicing, a crucial step to produce functional mRNA.

  • Eukaryotes add a 5’ cap (modified guanine nucleotide) and a 3’ poly-A tail (series of adenine nucleotides) to the transcript.

  • All mRNA processing events occur in the nucleus before export to the cytoplasm, ensuring precise control over gene expression.

Genes in Pieces
  • Introns: Noncoding, intervening sequences within a gene that are removed during RNA splicing. These can regulate gene expression and play a role in evolutionary processes.

  • Exons: Coding regions of a gene that are retained in the mature mRNA after splicing. They contain the instructions for protein synthesis.

  • mRNA genes can have anywhere from 0 to 362 introns, while tRNA genes have 0 or 1, showcasing the variability in gene structure.

RNA Splicing
  • Introns are transcribed along with exons in the primary transcript, forming a pre-mRNA molecule.

  • Splicing removes introns and joins exons to form mature RNA, which is then translated into protein.

    • Possibility 1: Introns are never transcribed - Polymerase somehow jumps from one exon to another (Incorrect mechanism).

    • Possibility 2: Introns are transcribed - Primary transcript results; an overlarge gene product is cut down by removing introns.

    • This is the correct process involving the spliceosome.

Splicing Signals
  • Splicing must be precise to maintain the correct reading frame and ensure proper protein synthesis.

  • Splicing signals in nuclear mRNA precursors are uniform. The first two bases of introns are GU, and the last two are AG. These are highly conserved.

    • 5’- and 3’-splice sites have consensus sequences with a branchpoint. These consensus sequences are crucial for spliceosome recognition and activity.

    • Yeast: 5’-AG/GUAAGU-intron-YNCURAC-YnNYAG/G-3’ (Y = pyrimidine, R = purine, N = any base). This is a typical yeast consensus sequence.

    • Whole consensus sequences are crucial for proper splicing. Even small mutations can disrupt splicing.

    • Mutations in consensus sequences can lead to abnormal splicing, resulting in non-functional or altered proteins. Examples include mutations causing genetic disorders.

Mechanism of Splicing of Nuclear mRNA Precursors
  • Splicing involves a branched intermediate called a lariat, which is a loop-like structure formed during the splicing process.

  • Two-step model:

    • The 2’-OH group of A in the middle of the intron attacks the phosphodiester bond between the first exon and the G at the beginning of the intron, forming the lariat. $ApGUAAGU.Thisisthefirsttransesterificationreaction.</p></li><li><p>The3OHleftattheendofthefirstexonattacksthephosphodiesterbondlinkingtheintronGtothesecondexon,formingtheexonexonphosphodiesterbond.. This is the first transesterification reaction.</p></li><li><p>The 3’-OH left at the end of the first exon attacks the phosphodiester bond linking the intron G to the second exon, forming the exon-exon phosphodiester bond.5’-AGG-3’.Thisisthesecondtransesterificationreaction.</p></li><li><p>Thisreleasestheintroninlariatform.Thelariatiseventuallydegraded.</p></li></ul></li></ul><h5id="4ac73e8d17ec4104aa4001b776be8353"datatocid="4ac73e8d17ec4104aa4001b776be8353"collapsed="false"seolevelmigrated="true">SignalattheBranch</h5><ul><li><p>Alongwithconsensussequencesat5and3endsofnuclearintrons,branchpointconsensussequencesalsooccur.Theseareessentialforlariatformation.</p></li><li><p>Yeastsequenceinvariant:UACUAAC(e.g.,yeastactingenestudy).Thissequenceishighlyconservedinyeast.</p></li><li><p>Highereukaryoteconsensussequenceismorevariable(U. This is the second transesterification reaction.</p></li><li><p>This releases the intron in lariat form. The lariat is eventually degraded.</p></li></ul></li></ul><h5 id="4ac73e8d-17ec-4104-aa40-01b776be8353" data-toc-id="4ac73e8d-17ec-4104-aa40-01b776be8353" collapsed="false" seolevelmigrated="true">Signal at the Branch</h5><ul><li><p>Along with consensus sequences at 5’- and 3’-ends of nuclear introns, branchpoint consensus sequences also occur. These are essential for lariat formation.</p></li><li><p>Yeast sequence invariant: UACUAAC (e.g., yeast actin gene study). This sequence is highly conserved in yeast.</p></li><li><p>Higher eukaryote consensus sequence is more variable (U{47}NN{C63}UU{53}RR{72}AA{91}CC{47}). The variability allows for more flexibility in splicing regulation.

    • Branched nucleotide is final A in the sequence. This A forms the 2’-5’ phosphodiester bond in the lariat structure.

    Spliceosomes
    • Splicing occurs on a particle called a spliceosome, a large RNA-protein complex.

    • Yeast and mammalian spliceosomes have sedimentation coefficients of 40S and 60S, respectively. These values indicate their size and complexity.

    • Spliceosomes contain pre-mRNA, snRNPs, and protein splicing factors. These components recognize key splicing signals and orchestrate the splicing process.

    snRNPs
    • Small nuclear RNAs coupled to proteins are abbreviated as snRNPs (snurps), small nuclear ribonuclear proteins. They are fundamental components of the spliceosome.

    • The snRNAs can be resolved on a gel: U1, U2, U4, U5, U6. Each snRNP has a specific role in splicing.

      • All 5 snurps join the spliceosome to play crucial roles in splicing. They recognize splice sites, catalyze splicing, and regulate the process.

    U1 snRNP
    • U1 snRNA sequence is complementary to both 5’- and 3’-splice site consensus sequences. This complementarity helps U1 bind to splice sites.

      • U1 snRNA base-pairs with these splice sites. It initiates the spliceosome assembly process.

      • Brings the sites together for splicing is too simple an explanation. The interaction is more complex and involves additional factors.

    • Splicing involves a branch within the intron.

    • Genetic experiments have shown that base pairing between U1 snRNA and 5’-splice site of mRNA precursor is necessary but not sufficient for binding. Alternative 5’-splice sites of E1A. Other factors are required for stable binding and commitment.

    U6 snRNP
    • U6 snRNP associates with the 5’-end of the intron by base pairing through the U6 RNA. This interaction is crucial for catalysis.

    • U6 cross-links to intron at +5, suggesting that the invariant sequence ACA in U6 base-pairs with the conserved UGU (+4 to +6) of the intron. This base pairing is essential for the first step in splicing.

    • Occurs first prior to the formation of the lariat intermediate but after the first step in splicing. U6 plays a catalytic role in the splicing process.

    • The association between U6 and splicing substrate is essential for the splicing process. It helps position the splice sites for the transesterification reactions.

    • U6 also associates with U2 during splicing (Active site). This interaction is central to the catalytic activity of the spliceosome.

    U2 snRNP
    • U2 snRNA base-pairs with the conserved sequence at the splicing branchpoint. This interaction is crucial for splicing. The branchpoint adenosine bulges out, facilitating the first transesterification reaction.

    • U2 (nuc 23 and 26-28) also forms base pairs with U6 (nuc 56-59):

      • This region is called helix I. It is a critical structural element in the spliceosome.

      • Helps orient snRNPs for splicing. Proper orientation is essential for catalysis.

    • 5’-end of U2 interacts with 3’-end of U6:

      • This interaction forms a region called helix II.

      • This region is important in splicing in mammalian cells, not in yeast cells. This highlights the differences in splicing mechanisms across different organisms.

    U5 snRNP
    • U5 snRNA shows no sequence complementarity but does associate with the last nucleotide in one exon and the first nucleotide of the next exon. It helps align the exons for splicing.

    • This should result in the two exons lining up for splicing. This is crucial for maintaining the correct reading frame.

    U4 snRNP
    • U4 base-pairs with U6 through stems I and II and does not play a direct role in splicing. Its role seems to be to bind and sequester U6.

    • When U6 is needed in a splicing reaction, U4 is removed to allow interaction with U2. This is a key regulatory step in spliceosome activation.

    • Interaction of U6 with U2 involves some U6 bases involved in U4 stem I formation. This highlights the dynamic interactions within the spliceosome.

    snRNP Involvement in mRNA Splicing
    • Spliceosomal complex contains (Substrate, U2, U5 & U6).

    • The complex ready for the 2nd step in splicing can be drawn as a group II intron (self-splicing intron) at the same stage of splicing. This suggests an evolutionary relationship between spliceosomes and group II introns.

    • Spliceosomal snRNPs substitute for elements at the center of catalytic activity of group II introns at the same stage of splicing. This further supports the evolutionary link.

    Spliceosome Catalytic Activity
    • Catalytic center of spliceosome appears to include Mg^{2+}andabasepairedcomplexof3RNAs:</p><ul><li><p>U2snRNA</p></li><li><p>U6snRNA(bindsand a base-paired complex of 3 RNAs:</p><ul><li><p>U2 snRNA</p></li><li><p>U6 snRNA (bindsMg^{2+}playsaroleincatalysis)</p></li><li><p>Branchpointregionoftheintron</p></li></ul></li><li><p>ProteinfreefragmentsoftheseRNAscancatalyzeareactionrelatedtothefirststepinsplicing.ThisunderscoresthecentralroleofRNAincatalysis.</p></li></ul><h5id="8ef94242038743989852c3182d43cac4"datatocid="8ef94242038743989852c3182d43cac4"collapsed="false"seolevelmigrated="true">SpliceosomeAssemblyandFunction</h5><ul><li><p>SpliceosomeiscomposedofmanycomponentsproteinsandRNA.Theprecisecompositionvariesdependingonthespecificsplicingevent.</p></li><li><p>Thesecomponentsassemblestepwise.Theassemblyprocessishighlyregulated.</p></li><li><p>Thespliceosomecycleincludesassembly,splicingactivity,anddisassembly.Thiscycleensuresefficientandaccuratesplicing.</p></li><li><p>Bycontrollingassemblyofthespliceosome,acellcanregulatethequalityandquantityofsplicingandsoregulategeneexpression.Thisisakeymechanismforcontrollingproteinproduction.</p></li></ul><h5id="f30fd82912ad47fda638ed925fef9f3d"datatocid="f30fd82912ad47fda638ed925fef9f3d"collapsed="false"seolevelmigrated="true">SpliceosomeCycle</h5><ul><li><p>AssemblybeginswithbindingofU1tosplicingsubstrateformingacommitmentcomplex(CC),aunitcommittedtosplicingouttheintron.</p></li><li><p>U2joins,withhelpfromATP,toformtheAcomplex.ATPhydrolysisprovidestheenergyforconformationalchanges.</p></li><li><p>Next,U4U6andU5jointoformtheB1complex.</p></li><li><p>U6dissociatesfromU4,anddisplacesU1atthe5splicesite:</p><ul><li><p>ThisstepisATPdependent.ATPhydrolysisdrivestheactivationofthespliceosome.</p></li><li><p>Activatesthespliceosome</p></li><li><p>AllowsU1andU4tobereleased</p></li><li><p>AllowsU6tobasepairwithU2</p></li></ul></li><li><p>ActivatedspliceosomeiscalledtheB2complex.</p></li><li><p>ATPprovidestheenergytoallowlariatformationintheC1complex.Thefirsttransesterificationreactionoccurs.</p></li><li><p>OnceanotherATPisconsumed,thesecondsplicingstepoccursresultingintheC2complex.Theexonsarejoined,andtheintronisreleased.</p></li><li><p>MatureRNAexitsthecomplex,leavingtheintronboundtotheIcomplex.Theintronisdegraded.</p></li></ul><h5id="d1710806aeaa43918b827a1297ca4db3"datatocid="d1710806aeaa43918b827a1297ca4db3"collapsed="false"seolevelmigrated="true">AMinorSpliceosome</h5><ul><li><p>Aminorclassofintronswithvariantbuthighlyconserved5splicesitesandbranchpointscanbesplicedwiththehelpofavariantclassofsnRNAs.Theseintronsarelesscommonbutimportantforspecificgenes.</p></li><li><p>CellscancontainaminorspliceosomewithminorsnRNAs:</p><ul><li><p>U11performslikeU1</p></li><li><p>U12actslikeU2</p></li><li><p>U4atacandU6atacperformlikeU4andU6,respectively</p></li><li><p>UsethesameU5asthemajorspliceosome</p></li></ul></li></ul><h5id="918202578a084bf797e58212bfaf0fc4"datatocid="918202578a084bf797e58212bfaf0fc4"collapsed="false"seolevelmigrated="true">Commitment,SpliceSiteSelection,andAlternativeSplicing</h5><ul><li><p>snRNPsdonothaveenoughspecificityandaffinitytobindexclusivelyandtightlyatexonintronboundaries.Additionalfactorsareneededforpreciserecognition.</p></li><li><p>AdditionalsplicingfactorsareneededtohelpsnRNPsbind.Thesefactorsenhancethespecificityandstabilityofspliceosomebinding.</p></li><li><p>SomesplicingfactorsareneededtobridgeacrossintronsandexonsandsodefinetheseRNAelements.Thesebridgingproteinsplayacrucialroleindefiningthesplicingboundaries.</p></li></ul><h5id="b905d24c48544b29844004ffc52e223c"datatocid="b905d24c48544b29844004ffc52e223c"collapsed="false"seolevelmigrated="true">ExonandIntronDefinition</h5><ul><li><p>Thespliceosomecanrecognizeeitherexonsorintronsinthesplicingcommitmentprocess,presumablybyassemblingsplicingfactorstobridgeacrossexonsorintrons.Themodeofrecognitionvariesbetweenorganisms.</p></li><li><p>Ifexonsarerecognized,itisexondefinition(highereukaryotes).SRproteinstypicallymediatethisprocess.</p></li><li><p>Ifintronsarerecognized,itisintrondefinition(yeast).BBPandMud2pareinvolvedinthisprocess.</p></li><li><p>Splicinginagivenorganismtypicallyuseseitherexondefinitionorintrondefinition.Thisiscriticalforpropersplicing.</p></li></ul><h5id="8c0108e9ee7949ad816e4b80374d1426"datatocid="8c0108e9ee7949ad816e4b80374d1426"collapsed="false"seolevelmigrated="true">Commitment</h5><ul><li><p>CommitmenttospliceatagivensiteisdeterminedbyanRNAbindingprotein.Thisisacriticalregulatorystep.</p></li><li><p>Thisproteinbindstothesplicingsubstrateandrecruitsotherspliceosomalcomponents.Thisinitiatesthespliceosomeassembly.</p></li><li><p>ThefirstcomponenttofollowisU1.U1bindingisoftentheinitialstepincommitment.</p></li><li><p>SRproteinsSC35andSF2/ASFcommitsplicingonhumanβglobinpremRNAandHIVtatpremRNA,respectively.TheseSRproteinsarespecifictocertainpremRNAs.</p></li><li><p>PartofthecommitmentinvolvestheattractionofU1insomecases.U1helpsstabilizetheinitialcomplex.</p></li><li><p>CommitmentwithdifferentpremRNAsrequiresdifferentsplicingfactors.Thisallowsforprecisecontrolofsplicing.</p></li></ul><h5id="34bfe6eb66f74efea318fa199a462fc7"datatocid="34bfe6eb66f74efea318fa199a462fc7"collapsed="false"seolevelmigrated="true">BridgingProteinsandCommitment</h5><ul><li><p>BecauseU1isthefirsttobind,interactingproteinsweresoughtout(syntheticlethalityinU1MUD2;inMUD2MSL5[BBP]).Theseproteinshelpstabilizethecomplex.</p></li><li><p>Yeastcommitmentcomplexhasabranchpointbridgingprotein(BBP)thatbindsto:</p><ul><li><p>U1snRNPproteinatthe5endoftheintron</p></li><li><p>Mud2pnearthe3endoftheintron</p></li><li><p>RNAnearthe3endoftheintron</p></li></ul></li><li><p>Bridgestheintronandcouldplayaroledefiningtheintronpriortosplicing.Thisbridginghelpsdefinethesplicingboundaries.</p></li><li><p>MammalianBBPisSF1,andMud2pisU2AF65andmayservethesamebridgingfunctiontodefineexons.Thismechanismisconservedacrosseukaryotes.</p></li></ul><h5id="b72e312cc3ff4d20a49793a40105ad2b"datatocid="b72e312cc3ff4d20a49793a40105ad2b"collapsed="false"seolevelmigrated="true">3SpliceSiteSelection</h5><ul><li><p>SplicingfactorSlu7isrequiredforcorrect3splicingsiteselection(properAGatthe3ofintron).Slu7ensuresthatthecorrectAGdinucleotideisused.</p></li><li><p>WithoutSlu7,splicingtothecorrect3splicesiteAGissuppressed,andsplicingtotheaberrantAGisactivated.Thiscanleadtononfunctionalproteins.</p></li><li><p>U2AFisalsorequiredfor3splicesiterecognition.U2AFisaheterodimerthatbindstothepolypyrimidinetractandAGdinucleotide.</p></li><li><p>65KDaU2AFsubunitbindstopolypyrimidinetractupstreamof3splicesiteand35KDasubunitbindstothe3splicesiteAG.Thisinteractionisessentialforproper3splicesiteselection.</p></li></ul><h5id="df9bd19f24e84829945f4443233965e6"datatocid="df9bd19f24e84829945f4443233965e6"collapsed="false"seolevelmigrated="true">RoleoftheRNAPolymeraseIICTD</h5><ul><li><p>CterminaldomainoftheRpb1subunitofRNApolymeraseIIstimulatessplicingofsubstratesthatuseexondefinition.TheCTDcoordinatestranscriptionandsplicing.</p></li><li><p>Thisdoesnotapplytothosethatuseintrondefinitiontoprepareforsplicing.Differentmechanismsareusedfordifferentmodesofsplicing.</p></li><li><p>CTDbindstosplicingfactorsandcouldassemblethefactorsattheendofexonstosetthemoffforsplicing.Thisassemblyiscrucialforefficientsplicing.</p></li></ul><h5id="dc1359b6e0ae4f4da1103c28be03512e"datatocid="dc1359b6e0ae4f4da1103c28be03512e"collapsed="false"seolevelmigrated="true">AlternativeSplicing</h5><ul><li><p>Transcriptsofmanyeukaryoticgenesaresubjecttoalternativesplicing.Thisgreatlyincreasesthediversityofproteinproducts.</p><ul><li><p>Thissplicingcanhaveprofoundeffectsontheproteinproductsofagene.</p></li><li><p>Canmakeadifferencebetween:</p><ul><li><p>Secretedormembraneboundprotein(µsandµm).Alternativesplicingcancontrolproteinlocalization.</p></li><li><p>Activityandinactivity.Alternativesplicingcanregulateproteinfunction.</p></li></ul></li><li><p>Productsof3genesinthesexdeterminationpathwayofthefruitflyaresubjecttoalternativesplicing.Thisisaclassicexampleofalternativesplicingregulation.</p></li></ul></li><li><p>Femalespecificsplicingofthetratranscriptgives:</p><ul><li><p>AnactiveproductthatcausesfemalespecificsplicingofdsxpremRNA.</p></li><li><p>Thisproducesafemalefruitfly.</p></li></ul></li><li><p>Malespecificsplicingofthetratranscriptgives:</p><ul><li><p>AninactiveproductthatallowsmalespecificsplicingofdsxpremRNA.</p></li><li><p>Thisproducesamalefruitfly.</p></li></ul></li><li><p>AlternativesplicingofthesamepremRNAgivesrisetoverydifferentproducts.</p><ul><li><p>Alternativesplicingpatternsoccurinoverhalfofhumangenes.Itisawidespreadphenomenon.</p></li><li><p>Manygeneshavemorethan2splicingpatterns,somehavethousands.Thisgeneratesimmenseproteindiversity.</p></li><li><p>Wehaveplays a role in catalysis)</p></li><li><p>Branchpoint region of the intron</p></li></ul></li><li><p>Protein-free fragments of these RNAs can catalyze a reaction related to the first step in splicing. This underscores the central role of RNA in catalysis.</p></li></ul><h5 id="8ef94242-0387-4398-9852-c3182d43cac4" data-toc-id="8ef94242-0387-4398-9852-c3182d43cac4" collapsed="false" seolevelmigrated="true">Spliceosome Assembly and Function</h5><ul><li><p>Spliceosome is composed of many components – proteins and RNA. The precise composition varies depending on the specific splicing event.</p></li><li><p>These components assemble stepwise. The assembly process is highly regulated.</p></li><li><p>The spliceosome cycle includes assembly, splicing activity, and disassembly. This cycle ensures efficient and accurate splicing.</p></li><li><p>By controlling assembly of the spliceosome, a cell can regulate the quality and quantity of splicing and so regulate gene expression. This is a key mechanism for controlling protein production.</p></li></ul><h5 id="f30fd829-12ad-47fd-a638-ed925fef9f3d" data-toc-id="f30fd829-12ad-47fd-a638-ed925fef9f3d" collapsed="false" seolevelmigrated="true">Spliceosome Cycle</h5><ul><li><p>Assembly begins with binding of U1 to splicing substrate forming a commitment complex (CC), a unit committed to splicing out the intron.</p></li><li><p>U2 joins, with help from ATP, to form the A complex. ATP hydrolysis provides the energy for conformational changes.</p></li><li><p>Next, U4-U6 and U5 join to form the B1 complex.</p></li><li><p>U6 dissociates from U4, and displaces U1 at the 5’-splice site:</p><ul><li><p>This step is ATP-dependent. ATP hydrolysis drives the activation of the spliceosome.</p></li><li><p>Activates the spliceosome</p></li><li><p>Allows U1 and U4 to be released</p></li><li><p>Allows U6 to base-pair with U2</p></li></ul></li><li><p>Activated spliceosome is called the B2 complex.</p></li><li><p>ATP provides the energy to allow lariat formation in the C1 complex. The first transesterification reaction occurs.</p></li><li><p>Once another ATP is consumed, the second splicing step occurs resulting in the C2 complex. The exons are joined, and the intron is released.</p></li><li><p>Mature RNA exits the complex, leaving the intron bound to the I complex. The intron is degraded.</p></li></ul><h5 id="d1710806-aeaa-4391-8b82-7a1297ca4db3" data-toc-id="d1710806-aeaa-4391-8b82-7a1297ca4db3" collapsed="false" seolevelmigrated="true">A Minor Spliceosome</h5><ul><li><p>A minor class of introns with variant but highly conserved 5’-splice sites and branchpoints can be spliced with the help of a variant class of snRNAs. These introns are less common but important for specific genes.</p></li><li><p>Cells can contain a minor spliceosome with minor snRNAs:</p><ul><li><p>U11 performs like U1</p></li><li><p>U12 acts like U2</p></li><li><p>U4atac and U6atac perform like U4 and U6, respectively</p></li><li><p>Use the same U5 as the major spliceosome</p></li></ul></li></ul><h5 id="91820257-8a08-4bf7-97e5-8212bfaf0fc4" data-toc-id="91820257-8a08-4bf7-97e5-8212bfaf0fc4" collapsed="false" seolevelmigrated="true">Commitment, Splice Site Selection, and Alternative Splicing</h5><ul><li><p>snRNPs do not have enough specificity and affinity to bind exclusively and tightly at exon-intron boundaries. Additional factors are needed for precise recognition.</p></li><li><p>Additional splicing factors are needed to help snRNPs bind. These factors enhance the specificity and stability of spliceosome binding.</p></li><li><p>Some splicing factors are needed to bridge across introns and exons and so define these RNA elements. These bridging proteins play a crucial role in defining the splicing boundaries.</p></li></ul><h5 id="b905d24c-4854-4b29-8440-04ffc52e223c" data-toc-id="b905d24c-4854-4b29-8440-04ffc52e223c" collapsed="false" seolevelmigrated="true">Exon and Intron Definition</h5><ul><li><p>The spliceosome can recognize either exons or introns in the splicing commitment process, presumably by assembling splicing factors to bridge across exons or introns. The mode of recognition varies between organisms.</p></li><li><p>If exons are recognized, it is exon definition (higher eukaryotes). SR proteins typically mediate this process.</p></li><li><p>If introns are recognized, it is intron definition (yeast). BBP and Mud2p are involved in this process.</p></li><li><p>Splicing in a given organism typically uses either exon definition or intron definition. This is critical for proper splicing.</p></li></ul><h5 id="8c0108e9-ee79-49ad-816e-4b80374d1426" data-toc-id="8c0108e9-ee79-49ad-816e-4b80374d1426" collapsed="false" seolevelmigrated="true">Commitment</h5><ul><li><p>Commitment to splice at a given site is determined by an RNA-binding protein. This is a critical regulatory step.</p></li><li><p>This protein binds to the splicing substrate and recruits other spliceosomal components. This initiates the spliceosome assembly.</p></li><li><p>The first component to follow is U1. U1 binding is often the initial step in commitment.</p></li><li><p>SR proteins SC35 and SF2/ASF commit splicing on human β-globin pre-mRNA and HIV tat pre-mRNA, respectively. These SR proteins are specific to certain pre-mRNAs.</p></li><li><p>Part of the commitment involves the attraction of U1 in some cases. U1 helps stabilize the initial complex.</p></li><li><p>Commitment with different pre-mRNAs requires different splicing factors. This allows for precise control of splicing.</p></li></ul><h5 id="34bfe6eb-66f7-4efe-a318-fa199a462fc7" data-toc-id="34bfe6eb-66f7-4efe-a318-fa199a462fc7" collapsed="false" seolevelmigrated="true">Bridging Proteins and Commitment</h5><ul><li><p>Because U1 is the first to bind, interacting proteins were sought out (synthetic lethality in U1 → MUD2; in MUD2 → MSL-5 [BBP]). These proteins help stabilize the complex.</p></li><li><p>Yeast commitment complex has a branchpoint bridging protein (BBP) that binds to:</p><ul><li><p>U1 snRNP protein at the 5’-end of the intron</p></li><li><p>Mud2p near the 3’-end of the intron</p></li><li><p>RNA near the 3’-end of the intron</p></li></ul></li><li><p>Bridges the intron and could play a role defining the intron prior to splicing. This bridging helps define the splicing boundaries.</p></li><li><p>Mammalian BBP is SF1, and Mud2p is U2AF65 and may serve the same bridging function to define exons. This mechanism is conserved across eukaryotes.</p></li></ul><h5 id="b72e312c-c3ff-4d20-a497-93a40105ad2b" data-toc-id="b72e312c-c3ff-4d20-a497-93a40105ad2b" collapsed="false" seolevelmigrated="true">3’-Splice Site Selection</h5><ul><li><p>Splicing factor Slu7 is required for correct 3’- splicing site selection (proper AG at the 3’ of intron). Slu7 ensures that the correct AG dinucleotide is used.</p></li><li><p>Without Slu7, splicing to the correct 3’-splice site AG is suppressed, and splicing to the aberrant AG is activated. This can lead to non-functional proteins.</p></li><li><p>U2AF is also required for 3’- splice site recognition. U2AF is a heterodimer that binds to the polypyrimidine tract and AG dinucleotide.</p></li><li><p>65KDa U2AF subunit binds to polypyrimidine tract upstream of 3’-splice site and 35KDa subunit binds to the 3’-splice site AG. This interaction is essential for proper 3’ splice site selection.</p></li></ul><h5 id="df9bd19f-24e8-4829-945f-4443233965e6" data-toc-id="df9bd19f-24e8-4829-945f-4443233965e6" collapsed="false" seolevelmigrated="true">Role of the RNA Polymerase II CTD</h5><ul><li><p>C-terminal domain of the Rpb1 subunit of RNA polymerase II stimulates splicing of substrates that use exon definition. The CTD coordinates transcription and splicing.</p></li><li><p>This does not apply to those that use intron definition to prepare for splicing. Different mechanisms are used for different modes of splicing.</p></li><li><p>CTD binds to splicing factors and could assemble the factors at the end of exons to set them off for splicing. This assembly is crucial for efficient splicing.</p></li></ul><h5 id="dc1359b6-e0ae-4f4d-a110-3c28be03512e" data-toc-id="dc1359b6-e0ae-4f4d-a110-3c28be03512e" collapsed="false" seolevelmigrated="true">Alternative Splicing</h5><ul><li><p>Transcripts of many eukaryotic genes are subject to alternative splicing. This greatly increases the diversity of protein products.</p><ul><li><p>This splicing can have profound effects on the protein products of a gene.</p></li><li><p>Can make a difference between:</p><ul><li><p>Secreted or membrane-bound protein (µs and µm). Alternative splicing can control protein localization.</p></li><li><p>Activity and inactivity. Alternative splicing can regulate protein function.</p></li></ul></li><li><p>Products of 3 genes in the sex determination pathway of the fruit fly are subject to alternative splicing. This is a classic example of alternative splicing regulation.</p></li></ul></li><li><p>Female-specific splicing of the tra transcript gives:</p><ul><li><p>An active product that causes female-specific splicing of dsx pre-mRNA.</p></li><li><p>This produces a female fruit fly.</p></li></ul></li><li><p>Male-specific splicing of the tra transcript gives:</p><ul><li><p>An inactive product that allows male-specific splicing of dsx pre-mRNA.</p></li><li><p>This produces a male fruit fly.</p></li></ul></li><li><p>Alternative splicing of the same pre-mRNA gives rise to very different products.</p><ul><li><p>Alternative splicing patterns occur in over half of human genes. It is a widespread phenomenon.</p></li><li><p>Many genes have more than 2 splicing patterns, some have thousands. This generates immense protein diversity.</p></li><li><p>We have2^6=64