Open AccessSafety and Feasibility of Liver-Directed Ex Vivo Gene Therapy for Homozygous Familial Hypercholesterolemia
Author(s) -
Steven E. Raper,
Mariann Grossman,
Daniel J. Rader,
Jess G. Thoene,
Bernard J. Clark,
Daniel M. Kolansky,
David W.M. Muller,
James M. Wilson
Publication year - 1996
Publication title -
annals of surgery
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 4.153
H-Index - 309
eISSN - 1528-1140
pISSN - 0003-4932
DOI - 10.1097/00000658-199602000-00002
Subject(s) - medicine , familial hypercholesterolemia , ex vivo , genetic enhancement , gene , genetics , bioinformatics , in vivo , cholesterol , biology
The purpose of this report was to provide detailed information on the safety and feasibility of surgical procedures associated with the first ex vivo liver-directed gene therapy trial for the treatment of vivo gene therapy for homozygous familial hypercholesterolemia (FH).