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

Mol Ther Methods Clin Dev. 2021-05; 
Lili Wang, Ilayaraja Muthuramu, Suryanarayan Somanathan, Hong Zhang, Peter Bell, Zhenning He, Hongwei Yu, Yanqing Zhu, Anna P Tretiakova, James M Wilson
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Molecular Biology Tools GeneScript Rare Codon Analysis Tool (https://www.genscript.com/tools/rare-codon-analysis) was used to evaluate the properties of the hLDLR-WT and optimized hLDLR cDNA that include CAI Get A Quote

摘要

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 ( , double knockout [DKO]). This study investigated vector efficacy following the combination of intervening sequence 2 (IVS2) of the human beta-globin gene and codon optimization with the previously developed gain-of-function, human LDLR triple-mutant variant (hLDLR-L318D/K809R/C818A) in the treatment of homozygous familial hypercholesterolemia (HoFH)... More

关键词

AAV8, HoFH, LDL, LDLR, adeno-associated virus, codon optimization, familial hypercholesterolemia, gene therapy, liver-directed