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Article

Non-Clinical Study Examining AAV8.TBG.hLDLR Vector-Associated Toxicity in Chow-Fed Wild-Type and LDLR+/- Rhesus Macaques.

Human gene therapy. Clinical development - 1 Mar 2017

Greig Jenny A, Limberis Maria P, Bell Peter, Chen Shu-Jen, Calcedo Roberto, Rader Daniel J, Wilson James M

Abstract excerpt

Vectors based on adeno-associated virus serotype 8 (AAV8) have been evaluated in several clinical trials of gene therapy for hemophilia B with encouraging results. In preparation for a Phase 1 clinical trial of AAV8 gene therapy for the treatment of homozygous familial hypercholesterolemia (HoFH), the safety of the clinical candidate vector, AAV8.TBG.hLDLR, was evaluated in wild-type rhesus macaques and macaques...

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Share to APOBEC-1 Deaminase
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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 28319449
DOI: 10.1089/humc.2017.014
PMCID: PMC5369385

Genes

LDLR

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