Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B

Adeno-associated virus (AAV) vectors are a leading platform for gene-based therapies for both monogenic and complex acquired disorders. The success of AAV gene transfer highlights the need to answer outstanding clinical questions of safety, durability, and the nature of the human immune response to...

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發表在:Mol Ther
Main Authors: George, Lindsey A., Ragni, Margaret V., Rasko, John E.J., Raffini, Leslie J., Samelson-Jones, Benjamin J., Ozelo, Margareth, Hazbon, Maria, Runowski, Alexa R., Wellman, Jennifer A., Wachtel, Katie, Chen, Yifeng, Anguela, Xavier M., Kuranda, Klaudia, Mingozzi, Federico, High, Katherine A.
格式: Artigo
語言:Inglês
出版: American Society of Gene & Cell Therapy 2020
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Original Article
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC7474338/
https://ncbi.nlm.nih.gov/pubmed/32559433
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2020.06.001
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