Developing a second-generation clinical candidate AAV vector for gene therapy of familial hypercholesterolemia

Gene therapy for hypercholesterolemia offers the potential to sustainably ameliorate disease for life with a single dose. In this study, we demonstrate the combinatorial effects of codon and vector optimization, which significantly improve the efficacy of an adeno-associated virus (AAV) vector in th...

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Detalles Bibliográficos
Autores principales: Wang, Lili, Muthuramu, Ilayaraja, Somanathan, Suryanarayan, Zhang, Hong, Bell, Peter, He, Zhenning, Yu, Hongwei, Zhu, Yanqing, Tretiakova, Anna P., Wilson, James M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8237527/
https://www.ncbi.nlm.nih.gov/pubmed/34258325
http://dx.doi.org/10.1016/j.omtm.2021.04.017

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8237527/
https://www.ncbi.nlm.nih.gov/pubmed/34258325
http://dx.doi.org/10.1016/j.omtm.2021.04.017

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