1、韩韩 晓晓Generation of adeno-associated virus vectorEarly generations of adenoviral vector(replication defective)Gutless Adenoviral vector(Amplicon)Generation of retroviral vectorPseudotyped retroviral vectorViral vectortitersmanupilation of immunogenicityinfecting of tropism non-dividing cellsAdenoviru
2、s1011terrificvery highyesRetrovirus107goodlowonly lentivirusHerpesvirus107not so goodlowyesAAV107not so goodlowyes Death of 18-year old Jesse Gelsinger Liver disease:OTC deficiency(genetic disease)University of Pennsylvania High dose of adenoviral vector(E1 and E4 genesdeleted)carrying the normal co
3、py of OTC gene was administered Suspected cause of death-Toxicity of high titer adenoviral vector-High immunogenicity of adenoviral vector(an immune revolt)A case of leukemia in a SCID child treated with a retroviral vectorBubble boy disease(T cell deficiency)Overall quite successful,over 1000 peopl
4、es received retroviral gene therapy A French babys treated with retroviral vector 3 years agoA leukemia-like illness developed this summer.Nine other children treated same time show no sign of leukemiaBut the side effect isnt a big enough risk yet that genetic experiments for children with an often fatal immune disease should stopPeople receiving retroviral gene therapy should be warned about the risk of developing leukemia
