Disease-regulated transgene expression in the CNS

Question 1

Which 5 categories of viral vectors are used for CNS gene therapy?

Answer 1

1. AD5 (adenovirus-associated vector serotype 5)
2. AAV2 (adeno-associated viral vector 2 serotype 2)
3. HSV (herpes simplex virus)
4. HIV / FIV / EIAV (lentiviral vectors)¨
5. Oncoretroviral vectors (MLVs)

Question 2

When inserting a gene for therapeutic purposes, what should you take into extra consideration?

Answer 2

The gene should be downstream of a tissue-specific promoter. Examples: NSE for neurons, GFAP for glial cells. ¨