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
12/01/2013
Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression.
Mueller C, Chulay JD, Trapnell BC, Humphries M, Carey B, Sandhaus RA, McElvaney NG, Messina L, Tang Q, Rouhani FN, Campbell-Thompson M, Fu AD, Yachnis A, Knop DR, Ye GJ, Brantly M, Calcedo R, Somanathan S, Richman LP, Vonderheide RH, Hulme MA, Brusko TM, Wilson JM, Flotte TR
The Journal Of Clinical Investigation
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
