分子病毒学 8病毒载体(共页)

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 cellsAdenovirus1011terrificvery 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 copy 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 peoples 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