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Article

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

4 May 2021

Abstract excerpt

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 the low-density lipoprotein receptor (LDLR)-deficient mouse model ( Ldlr −/− , Apobec1 −/− double knockout [DKO])....

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PMID: 34258325
DOI: 10.1016/j.omtm.2021.04.017

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