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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Publicado en:Mol Ther
Autores principales: 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.
Formato: Artigo
Lenguaje:Inglês
Publicado: American Society of Gene & Cell Therapy 2020
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Original Article
Acceso en línea: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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