Genetic Analysis and Recombinant DNA Technology

Chapter 08 Genetic Analysis and Recombinant DNA Technology

Section 8.1: Tools and Techniques of Recombinant DNA Technology

  • Learning Outcomes:

    • Provide examples of practical applications of modern genetic technologies.

    • Explain the role of restriction endonucleases in the process of recombinant DNA technologies.

    • List the steps in the polymerase chain reaction (PCR) and discuss one disadvantage to this technique.

    • Describe how recombinant DNA is created and discuss its role in gene cloning.

Applications of Recombinant DNA Technology
  • From Basic Science:

    • Utilizing DNA to identify individuals.

    • Fixing underlying genetic mutations to treat diseases.

    • Using CRISPR technology to repair genetic mutations in a way that the fixes can be transferred to future generations.

DNA: The Raw Material
  1. Intrinsic Properties of DNA:

    • DNA strands separate when exposed to temperatures just below boiling.

    • When separated, the nucleotides are exposed allowing for identification, replication, and transcription of the DNA molecule.

    • Complementary nucleotides will hydrogen bond to each other, and the strands will regain their double-stranded form upon slow cooling.

  2. Heating and Cooling of DNA:

    • Heating causes DNA to denature (lose its hydrogen bonding) and separate into two strands.

    • When cooled, the two strands rejoin at complementary regions; they do not have to originate from the same organism as long as they have matching nucleotides.

Restriction Endonucleases
  • Function: Enzymes that clip DNA crosswise at selected locations.

    • Recognize foreign DNA to act upon.

    • Break phosphodiester bonds between adjacent nucleotides on both strands of DNA.

  • Each endonuclease recognizes sequences of 4 to 10 base pairs.

  • Protect bacteria and archaea against incompatible DNA from bacteriophages or plasmids.

Palindromes and Sticky Ends
  • Palindromic Sequences:

    • Sequences of DNA that are identical when read from the 5' to 3' direction on one strand and the 5' to 3' on the other strand.

  • Sticky Ends:

    • Staggered symmetrical cuts that leave short tails of 4 to 5 bases on each strand.

    • Base pair with complementary tails on other DNA fragments or plasmids.

Restriction Fragments and RFLPs
  • Restriction Fragments:

    • Pieces of DNA produced by restriction endonucleases.

  • Restriction Fragment Length Polymorphisms (RFLPs):

    • Differences in the cutting patterns of specific endonucleases resulting in restriction patterns of different lengths.

    • Allow direct comparison of DNA of two different organisms at specific sites.

Ligase and Reverse Transcriptase
  • Ligase:

    • Seals sticky ends together by rejoining the phosphate-sugar bonds cut by endonucleases.

    • Main application is the final splicing of genes into plasmids and chromosomes.

  • Reverse Transcriptase:

    • Enzyme that replicates HIV and other retroviruses, converting RNA into DNA.

Making cDNA From Eukaryotic mRNA
  • Complementary DNA (cDNA):

    • Made from messenger RNA (mRNA), transfer RNA (tRNA), ribosomal RNA (rRNA), and others.

    • Useful in synthesizing eukaryotic genes from mRNA transcripts, which are free of introns.

CRISPR Technology
  • CRISPR - Clustered Regularly Interspaced Short Palindromic Repeats:

    • Short lengths of DNA with repeating nucleotides found in bacteria and archaea.

    • Enzyme Cas9 cuts out foreign DNA left behind by invading bacteriophages or plasmids.

    • Scientists exploit this to cut DNA in virtually any organism precisely.

Polymerase Chain Reaction (PCR)
  • Function:

    • Rapidly increases the amount of DNA in a sample without the need for cultures or complex purification techniques.

    • Can replicate a target DNA from a few copies to billions in a few hours.

  • Sensitivity:

    • Can detect cancer from a single cell and diagnose infections from a single gene copy.

Steps of DNA Replication in PCR
  • Uses events of DNA replication:

    1. Opening of the double helix.

    2. Using exposed strands as templates.

    3. Addition of primers.

    4. Action of DNA polymerase.

Specialized Ingredients Used in PCR
  • Primers:

    • Synthetic oligonucleotides of a known sequence (15-30 bases) that indicate the start of DNA amplification.

  • DNA Polymerases:

    • Enzymes responsible for DNA replication.

    • High temperatures necessitate the use of DNA polymerases isolated from thermophilic bacteria like Taq polymerase from Thermus aquaticus and Vent polymerase from Thermococcus litoralis.

PCR Technique
  • Utilizes a thermal cycler performing cyclic temperature changes.

  • Three Basic Steps:

    1. Denaturation.

    2. Priming.

    3. Extension.

  • Cyclic repetition amplifies DNA exponentially (e.g., 68 billion molecules from one copy).

Additional PCR Techniques
  • Real-time PCR:

    • Detect products during the reaction instead of at endpoint.

  • Adaptation to Analyze RNA:

    • Reverse transcriptase converts RNA to cDNA which can be amplified with PCR.

  • Loop-mediated Isothermal Amplification (LAMP):

    • Does not go through temperature variations to complete PCR.

Methods in Recombinant DNA Technology
  • Definition:

    • Deliberately removing genetic material from one organism and combining it with another to form genetic clones.

  • Cloning Process:

    1. Removal of a gene from a genetic donor (animal, plant, or microorganism).

    2. Insertion of the gene into a vector (plasmid or virus) that will be inserted into a cloning host.

    3. Translation of the gene into its corresponding protein product.

Strategies for Obtaining Genes in Isolated State
  • Common Strategies:

    • Removal of DNA from cells using endonucleases to separate into fragments.

    • Synthesis from isolated mRNA transcripts using reverse transcriptase (to create cDNA).

    • Amplification of the gene using PCR.

Genomic Libraries
  • Definition:

    • Genes maintained in a cloning host and vector, similar to microbial pure culture.

    • Collections of cDNA clones representing the entire genome of several organisms.

Cloning Vectors
  • Plasmids:

    • Small, well-characterized, easy to manipulate, can be transferred into host cells through transformation, carry genetic markers for antibiotic resistance.

  • Bacteriophage:

    • Naturally capable of injecting DNA into bacterial hosts via transduction.

Important Aspects of Cloning Vectors
  • Origin of Replication (ORI):

    • Must be replicated by the DNA polymerase of the cloning host.

  • Size Acceptance:

    • Must accept DNA of the desired size.

    • Drug Resistance Gene:

    • Typically contain a gene that provides drug resistance to their cloning host.

Desirable Features in a Microbial Cloning Host
  • Characteristics:

    • Rapid turnover and fast growth rate.

    • Large-scale growth using standard culture methods.

    • Nonpathogenic.

    • In-depth genome mapping.

    • Capacity to accept plasmid or bacteriophage vectors.

    • Maintain foreign genes through multiple generations.

    • High yield of proteins from expressed foreign genes.

Example of Recombinant DNA Technology
  • Production of Alpha-2a Interferon (Roferon-A):

    • Used to treat hairy cell leukemia and Kaposi’s sarcoma.

    • Human alpha interferon gene codes for a polypeptide and is isolated from mRNA transcripts free of introns.

Ingredients for Gene Cloning
  • Essential Components:

    • The gene of interest (e.g., interferon) and a cloning vector (usually a plasmid).

First Steps in Gene Cloning
  • Procedure:

    • Digest the donor gene and plasmid using the same restriction enzyme, producing complementary sticky ends.

    • Base-pair the gene and plasmid together; ligase seals the bonds covalently.

    • Introduce the recombinant plasmid through transformation into the cloning host (lab strain of E. coli).

    • Use selective medium to identify recombinant clones.

Synthesis and Purification of Protein in Gene Cloning
  • As the cells multiply, each cell carries the plasmid along with its chromosome.

  • Once cloned and tested, the recombinant strain cultures without redoing the cloning step.

  • The donor gene is transcribed to mRNA and translated into the protein product.

Synthetic Biology
  • Definition:

    • Creation of new biological molecules and organisms from scratch.

    • In 2010, scientists created a self-replicating bacterium from DNA nucleotides.

    • Applications: Precise chemical synthesis to replace compounds missing in diseases, customized immune components, and targeting cancerous cells or pathogens.

Section 8.2: DNA Analysis

  • Learning Outcomes:

    • Describe how gel electrophoresis is used for DNA analysis.

    • Outline steps in DNA profiling.

    • Summarize the DNA sequencing process.

    • Discuss the significance of single nucleotide polymorphisms (SNPs) in DNA analysis.

    • Describe uses of microarray technology.

Genome Analysis: Maps and Profiles
  • Analysis of DNA reveals genetic abnormalities, ancestry, and determines if samples are the same.

  • Example: Identifying a criminal via DNA from a hair at a crime scene.

Gel Electrophoresis
  • Function:

    • Produces readable patterns of DNA fragments from samples subjected to an electric current in agar gel.

    • DNA, negatively charged from phosphate groups, moves toward the positive pole; speed depends on fragment size.

    • Staining allows for visualization of DNA fragment positions.

Distinctive Electrophoresis Patterns
  • Useful in characterizing DNA fragments and comparing genetic similarities across samples.

Nucleic Acid Hybridization and Probes
  • Hybridization:

    • Different nucleic acids unite at complementary regions; ssDNA can hybridize with other ssDNA or RNA.

Gene Probes
  • Definition:

    • Short DNA stretches of known sequences that base pair with complementary sequences in a sample.

    • Carry reporter molecules like fluorescent dyes for visualization of hybridization areas.

Uses of Gene Probes
  • Useful for diagnosing infections and identifying unknown bacterial or viral cultures.

Nucleic Acid Hybridization Test
  • Procedure that doesn’t require electrophoresis; DNA is isolated, denatured, and combined with specific probes for hybridization, subsequently developed and observed for hybridization areas.

Fluorescent In Situ Hybridization (FISH)
  • Probes applied to intact cells and microscopically observed locate specific genetic markers on genes.

    • Useful for identifying genes on chromosomes and bacteria without culturing.

    • Also detects RNA in cells/tissues.

Size of DNA Analysis
  • Relative sizes of nucleic acids denoted by base pairs (bp):

    • Palindromic sequences: 4 to 10 bp.

    • Average gene in E. coli: 1,300 bp or 1.3 kb.

    • Entire E. coli genome: 4,700,000 bp or 4.7 Mb.

    • Human mitochondrion: 16 kb; Epstein–Barr virus: 172 kb; Human genome: 6 billion bp (46 chromosomes).

DNA Profiles: A Unique Picture of the Genome
  • DNA Profiling:

    • Forensic tool relying on small unique differences in DNA between individuals (same species).

    • Restriction enzymes cut DNA, producing fragments of varying lengths in digested samples from different individuals.

Restriction Fragment Length Polymorphism (RFLP)
  • Function:

    • Restriction enzymes cut DNA at specific sites; whether the strand has the site determines if it is cut into pieces.

DNA Fingerprinting Dependence on RFLP
  • Involves digesting samples with restriction enzymes, separating using gel electrophoresis, leading to distinct banding patterns that depend on variations in DNA.

Sequence Maps
  • Function:

    • Detailed genome maps indicating base order in plasmids, chromosomes, or genomes.

    • Thousands of organisms sequenced; genetic similarities noted across species (e.g., humans share 80% DNA with mice).

Whole-Genome Shotgun Sequencing: Steps 1–4
  • Process involves breaking down whole genomes into smaller fragments, purifying, transforming into E. coli, sequencing clonal inserts, and assembling contigs.

Whole-Genome Shotgun Sequencing: Steps 5–7
  • Utilize computer programs to align sequences and construct larger contiguous sets (contigs), followed by human analysis for irregularities and ambiguities.

High-Throughput Genome Sequencing
  • Development of sophisticated systems for quicker genome sequencing with minimal human intervention.

Cost to Sequence a Human Genome (2001–2020)
  • Initially took 13 years & $3 billion; now can be completed in an afternoon at less than $1,000.

Genomics and Bioinformatics
  • Managing data from high-throughput sequencing, analyzing and classifying genes, determining protein sequences, and gene functions.

Annotating the Genome
  • Determines functional information of gene sequences, aiding in understanding cell function, disease, development, and aging.

Single Nucleotide Polymorphism (SNP)
  • Definition:

    • SNPs involve single nucleotide alterations, impacting individual traits, with 10 million SNPs mapped in the human genome; used to identify disease risks (e.g., thrombophilia from a mutation in the clotting factor V gene).

Example of SNP Associated with Disease
  • Normal vs. Excessive Clotting:

    • Normal: Codon GCT to CGA transcribes to Arginine (Arg), allowing proper clotting.

    • Excessive: Codon GTT to CAA changes Arginine to Glutamine (Gln), preventing inhibition and causing excessive clotting.

DNA Microarray Analysis
  • Allows biologists to monitor gene expression levels across thousands of genes in cells.

Uses of Microarrays
  • Developing diagnostic tests that target specific patterns of gene expression to identify cancer subtypes.

Section 8.3: Genetic Approaches to Healing Disease

  • Learning Outcomes:

    • Define personalized medicine.

    • Provide examples of recombinant products contributing to human health.

    • List recombinant bacteria and plants with purpose.

    • Differentiate somatic and germline gene therapy.

    • Describe the impact of miRNAs on human diseases.

    • Explain CRISPR-Cas9 usage for curing genetic diseases.

Products of Recombinant DNA Technology
  • Uses:

    • Producing recombinant organisms and protein products, nucleotide sequences, and medications that can’t be manufactured otherwise.

    • Large-scale hormone and enzyme manufacture like insulin & human growth hormone.

Current Medicines from Recombinant DNA Technology
  • Immune Treatments:

    • Interferons for cancer, MS, viral infections.

    • Interleukins for white blood cell regulation.

    • TNF for cancer treatment.

    • Biologics like Remicade® and Humira® for autoimmune disorders.

  • Hormonal Treatments:

    • Erythropoietin (EPO) for anemia; Human Growth Hormone (HGH) for growth issues; rhDNase for cystic fibrosis; tPA for dissolving blood clots; PEG-SOD to minimize brain and organ damage.

Vaccines and Additional Medicines
  • Vaccines for hepatitis B, HPV, Haemophilus influenzae type b; Factor VIII for hemophilia A.

Genetically Modified Organisms (GMOs)
  • Definition:

    • Transgenic organisms produced by integrating foreign genes to create recombinant microbes, plants, and animals.

Uses of Recombinant Microbes
  • - Modifying Pseudomonas syringae to prevent ice crystals; Pseudomonas fluorescens to destroy insects; making plants resistant to pests by inserting insecticide genes from Bacillus thuringiensis.

Controversy Surrounding Transgenic Plants
  • Concerns about gene sharing leading to “superweeds” versus the fact that genes swap in nature.

    • Inserting insect-resistance genes can decrease the need for pesticides.

Gene Therapy
  • Goal:

    • Repair or correct faulty genes for permanent cures of diseases.

Strategies for Gene Therapy
  • Cloning normal genes in retrovirus/adenovirus vectors, infecting patient tissues, reintroducing transfected cells, or using naked DNA for direct introduction into tissues.

Gene Therapy Treatments (As of 2022)
  • Approved for 13 diseases: e.g., spinal muscular atrophy, inherited vision loss, and acute lymphoblastic leukemia.

  • Somatic Cell Gene Therapy:

    • Changes are permanent in the individual but not heritable.

  • Germline Therapy:

    • Genes inserted into egg/sperm/embryos; changes are heritable.

Small RNAs as Medicine
  • miRNAs silence gene expression; introducing them can inhibit cancer growth in mice (e.g., lung and liver cancers).

  • Inhibiting miRNAs:

    • Stopping specific miRNAs can halt ovarian and breast cancers; lowering cholesterol; inhibit hepatitis C virus multiplication in chimpanzees.

Tests in Humans
  • The first successful human application of interfering RNAs involved nasal sprays with miRNA, showing reduced RSV infections in volunteers.

CAR-T Cell Treatment of Cancer
  • DNA recombination techniques modify T cells to battle cancer cells.

    • Patients’ T cells are altered to recognize and destroy cancer cells; currently an expensive treatment.

CRISPR-Cas9 System
  • A naturally occurring gene-editing system in bacteria; awarded the 2020 Nobel Prize in Chemistry for its precise gene editing capabilities.

  • Awardees: Dr. Jennifer Doudna and Dr. Emmanuelle Charpentier.

Concept Checks

  • Section 8.1:

    • Describe two scenarios for manipulating genetic information for human benefit.

    • DNA can be cut by enzymes called ____.

    • cDNA is produced using the enzyme ____.

    • True/False: PCR uses specialized DNA polymerase adapted to high temperatures.

    • List two cloning vectors and two cloning hosts.

  • Section 8.2:

    • Outline steps in whole-genome shotgun sequencing.

    • ____ genomes provide complete understanding of cell function and disease.

    • DNA profiling depends on ____ enzymes.

    • A(n) ____ allows scientists to view gene expression in cells.

  • Section 8.3:

    • List three products from recombinant DNA technology.

    • Recombinant organisms with foreign genes are called ____.

    • ____ therapy refers to gene therapy in eggs, sperm, or embryo.

    • True/False: Naked DNA is often used in gene therapy.

    • True/False: CRISPR-Cas9 naturally occurs in bacteria.