Gene Therapy Flashcards
What is Somatic gene therapy?
The process in which therapeutic genes are delivered into the somatic cells of a diseased patient.
What is Germline therapy?
The process in which germ cells or a zygote is genetically modified.
How has Somatic gene therapy been used to treat Severe Combined Immune Deficiency (SCID)?
A retrovirus has been used to deliver the gene encoding for ‘adenosine deaminase (ADA)’ to the target somatic cells of the patient.
What is the function of Adenosine deaminase (ADA)?
Adenosine deaminase is an enzyme involved in purine metabolism. and plays a role in development and maintenance of the immune system.
How does a lack of ADA result in Severe Combined Immune Deficiency?
A lack of ADA leads to the accumulation of dATP. dATP inhibits the activity of ribonucleotide reductase, which functions to reduce ribonucleotides to produce deoxyribonucleotides. As the immune system relies on cell proliferation, which requires DNA synthesis and thus, deoxyribonucleotides, it’s absence will affect the functioning of the immune system.
What are Zinc-finger nucleases (ZFNs)?
ZFNs are artificial restriction enzymes used to target specific desired DNA sequences to alter the genomes of higher organisms.
What are the three technologies used for targeted gene modification?
Zinc-Finger Nucleases (ZFNs), Transcription Activator-like Effector Nucleases (TALENs) and CRISPR/Cas9.
What co-receptor does HIV-1 use to infect its target cells?
HIV-1 uses the C-C chemokine receptor type 5 (CCR5).
What is the function of Dystrophin?
Dystrophin is essential for muscle cell membrane integrity. It does this by connecting the cytoskeleton of a muscle fiber to the surrounding ECM.
What is Dystrophin?
It is a protein complex that connects the cytoskeleton of a muscle fiber to the surrounding ECM [through the cell membrane].
