Characterization of the humanized FRG mouse model and development of an AAV-LK03 variant with improved liver lobular biodistribution

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. In the present study, we first investig...

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Detalles Bibliográficos
Autores principales: Cabanes-Creus, Marti, Navarro, Renina Gale, Liao, Sophia H.Y., Scott, Suzanne, Carlessi, Rodrigo, Roca-Pinilla, Ramon, Knight, Maddison, Baltazar, Grober, Zhu, Erhua, Jones, Matthew, Denisenko, Elena, Forrest, Alistair R.R., Alexander, Ian E., Tirnitz-Parker, Janina E.E., Lisowski, Leszek
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9860073/
https://www.ncbi.nlm.nih.gov/pubmed/36700121
http://dx.doi.org/10.1016/j.omtm.2022.12.014

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9860073/
https://www.ncbi.nlm.nih.gov/pubmed/36700121
http://dx.doi.org/10.1016/j.omtm.2022.12.014

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