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
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