Gene Therapy Lecture Notes

Flashcards covering different aspects of gene therapy. Including somatic cell gene therapy, vectors and RNA modification.

What is gene therapy?

Involves transferring DNA and or RNA into patient cells to modify gene expression or correct/compensate for an abnormal allele.

What is somatic cell gene therapy?

Adding a gene into the affected individual, typically for recessive diseases to supply a functioning copy of the defective gene.

What is RNA modification?

Targets pathogenic mutant genes (dominant mutations) by correcting or removing transcripts from the disease-causing allele.

What are somatic cells?

The cells of the body.

What is germline gene therapy?

Affects the germ cells, leading to a permanent and transmissible genetic modification affecting offspring.

What is the goal of somatic cell gene therapy?

Genetic modification for the purpose of curing disease in the correct tissues, by inserting a wild type allele.

What is ex vivo gene therapy?

Involves removing cells from the body, modifying them genetically in culture, and then reintroducing them back into the patient.

What is in vivo gene therapy?

Involves delivering DNA directly into the patient's cells in vivo, targeting the affected tissue or circulation.

What are vectors in gene therapy?

Systems used to carry and introduce DNA into cells, avoiding immune responses.

What are integrating vectors?

Incorporate into the host genome, for long term expression.

What are episomal vectors?

Carry DNA into the nucleus for expression but do not integrate, reducing the risk of mutations.

What are oncoretroviral vectors?

RNA based viruses that reverse transcribe into cDNA and integrate into the host genome randomly.

What are adenoviral vectors?

Cause benign infections of the upper respiratory tract and can efficiently transduce dividing and non dividing cells.

What are adeno associated viral vectors (AAV)?

An integrating vector that integrates at a specific position in the genome without interfering with endogenous genes.

What is a lentivirus?

A type of retrovirus that can infect non dividing cells but poses safety concerns due to its hazardous nature.

What are liposomes?

Lipid vesicles that encapsulate and protect DNA for delivery into cells.

What is a gene gun?

A technique in plants that involves firing particles coated in DNA into cells.

What is receptor mediated endocytosis?

Enhances uptake of molecules by coupling a protein to the surface of a particle that recognizes a specific cell type.

What is severe compromised immune deficiency (SCID)?

An X linked condition with defective BNT lymphocytes, leading to severely compromised immune systems.

What is antisense oligotherapy?

The use of oligos to rescue the product of the SMN2 gene to replace the missing SMN1 gene in spinal muscular atrophy.

What are ribozymes?

Short RNA sequences that cleave and bind to a target gene using complementary base pairing.

What are antisense oligonucleotides?

Small RNA molecules that bind complementary to a splice sequence, blocking splicing.

What is RNA interference (RNAi)?

Involves making a double stranded RNA with a hairpin sequence that is cleaved by dicer, targeting mRNA for degradation.