We invite you to discover Human Gene Therapy Clinical Development, a peer-reviewed journal publishing preclinical research and clinical trial data for gene and cell therapies.

Publication 3/01/2017

Nonclinical Pharmacology/Toxicology Study of AAV8.TBG.mLDLR and AAV8.TBG.hLDLR in a Mouse Model of Homozygous Familial Hypercholesterolemia.

Greig JA, Limberis MP, Bell P, Chen SJ, Calcedo R, Rader DJ, Wilson JM

Human Gene Therapy. Clinical Development

Publication 3/01/2017

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

Greig JA, Limberis MP, Bell P, Chen SJ, Calcedo R, Rader DJ, Wilson JM

Human Gene Therapy Clinical Development

Publication 5/01/2016

Stable liver-specific expression of human IDOL in humanized mice raises plasma cholesterol.

Ibrahim S, Somanathan S, Billheimer J, Wilson JM, Rader DJ

Cardiovascular Research

Publication 8/29/2014

AAV vectors expressing LDLR gain-of-function variants demonstrate increased efficacy in mouse models of familial hypercholesterolemia.

Somanathan S, Jacobs F, Wang Q, Hanlon AL, Wilson JM, Rader DJ

Circulation Research

Publication 12/01/2013

Biodistribution of AAV8 vectors expressing human low-density lipoprotein receptor in a mouse model of homozygous familial hypercholesterolemia.

Chen SJ, Sanmiguel J, Lock M, McMenamin D, Draper C, Limberis MP, Kassim SH, Somanathan S, Bell P, Johnston JC, Rader DJ, Wilson JM

Human Gene Therapy. Clinical Development

Publication 1/01/2013

Adeno-associated virus serotype 8 gene therapy leads to significant lowering of plasma cholesterol levels in humanized mouse models of homozygous and heterozygous familial hypercholesterolemia.

Kassim SH, Li H, Bell P, Somanathan S, Lagor W, Jacobs F, Billheimer J, Wilson JM, Rader DJ

Human Gene Therapy