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...

Täydet tiedot

Tallennettuna:
Bibliografiset tiedot
Julkaisussa:Mol Ther Methods Clin Dev
Päätekijät: Wang, Lili, Muthuramu, Ilayaraja, Somanathan, Suryanarayan, Zhang, Hong, Bell, Peter, He, Zhenning, Yu, Hongwei, Zhu, Yanqing, Tretiakova, Anna P., Wilson, James M.
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: American Society of Gene & Cell Therapy 2021
Aiheet:
Original Article
Linkit:https://ncbi.nlm.nih.gov/pmc/articles/PMC8237527/
https://ncbi.nlm.nih.gov/pubmed/34258325
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2021.04.017
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