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Genetics (David Oh) > Gene Therapy > Flashcards

Flashcards in Gene Therapy Deck (12):

retroviral vectors

advantage that they integrate their genomes into the host genome, so allowing for long-term persistence and gene expression. Historically the most popular


lentiviral vectors

are a subset, derived from HIV-1, with broader host cell range – very good at delivering genes to cells of hematopoietic system


adenoviral vectors

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


e.g. suicide gene therapy

herpes simplex virus thymidine kinase (TK)