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Novel human liver-tropic AAV variants define transferable domains that markedly enhance the human tropism of AAV7 and AAV8

Recent clinical successes have intensified interest in using adeno-associated virus (AAV) vectors for therapeutic gene delivery. The liver is a key clinical target, given its critical physiological functions and involvement in a wide range of genetic diseases. Here, we report the bioengineering of a...

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Detalles Bibliográficos
Autores principales: Cabanes-Creus, Marti, Navarro, Renina Gale, Zhu, Erhua, Baltazar, Grober, Liao, Sophia H.Y., Drouyer, Matthieu, Amaya, Anais K., Scott, Suzanne, Nguyen, Loan Hanh, Westhaus, Adrian, Hebben, Matthias, Wilson, Laurence O.W., Thrasher, Adrian J., Alexander, Ian E., Lisowski, Leszek
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8693155/
https://www.ncbi.nlm.nih.gov/pubmed/34977275
http://dx.doi.org/10.1016/j.omtm.2021.11.011