Adeno-associated virus(AAV)is one of the best viral vectors for the drug delivery in gene therapy.Several U.S.Food and Drug Administration(FDA)proved drugs such as Luxturna,Zolgensma,Hemgenix or Elevidys are based on ...Adeno-associated virus(AAV)is one of the best viral vectors for the drug delivery in gene therapy.Several U.S.Food and Drug Administration(FDA)proved drugs such as Luxturna,Zolgensma,Hemgenix or Elevidys are based on wild-type AAV2,AAV9,AAV5 or AAVrh74,respectively.However,the highly liver bio-distribution and tropism generate high toxicity which leads to serious adverse events(SAE)or deaths in many clinical cases.展开更多
文摘Adeno-associated virus(AAV)is one of the best viral vectors for the drug delivery in gene therapy.Several U.S.Food and Drug Administration(FDA)proved drugs such as Luxturna,Zolgensma,Hemgenix or Elevidys are based on wild-type AAV2,AAV9,AAV5 or AAVrh74,respectively.However,the highly liver bio-distribution and tropism generate high toxicity which leads to serious adverse events(SAE)or deaths in many clinical cases.
