Flashcards in Gene Therapy Deck (12):
advantage that they integrate their genomes into the host genome, so allowing for long-term persistence and gene expression. Historically the most popular
are a subset, derived from HIV-1, with broader host cell range – very good at delivering genes to cells of hematopoietic system
not permanent (can be good thing), but easy to make in large amounts. However, can provoke an immune reaction. ie. common cold
adeno-associated viral (AAV) vectors
long lasting even though don’t integrate, seem to be non-immunogenic, increasingly popular
what is the difference between a retrovirus and a retroviral vector
retroviral vectors transcribe vector RNA genomes that ONLY code for the THERAPEUTIC gene. no viral proteins are made
what are the 5 examples of gene therapies
gene addition, localized gene delivery, gene knockout, Cancer - CARs, Cancer - suicide gene therapy
what is gene addition typically used for?
x linked SCID, ADA deficiency, genetic diseases. treated by adding the missing gene to T cells or HSC
what is localized gene delivery typically used for?
AAV vectors and Leber's congenital amaurosis
what do leukemic cells have?
integrated retroviral vectors near the LMO-2 gene
what is the inherited form of progressive blindness caused by and how can it be fixed?
defective RPE65. inject normal copy carried by an AAV vector
what are the 7 gene therapy approaches to cancer
1. replace a missing tumor suppressor gene
2. block expression of an oncogene
3. insert "suicide genes" into tumors
4. insert genes to induce anti-tumor immune responses
5. express genes which impede tumor neo-vasculature
6. add chemotherapy resistance genes to HSC to allow chemo intesification
7. chimeric antigen receptors CARs