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. Author manuscript; available in PMC: 2012 Apr 12.
Published in final edited form as: Clin Lipidol. 2010 Jun;5(6):793–809. doi: 10.2217/clp.10.73

Table 1.

Clinical trials involving gene replacement and gene inhibition for dyslipidemia conducted to date.

Disease Platform/name of therapy Gene target Primary sponsor Status of trial/clinical trial number Ref.
HoFH Ex vivo gene therapy LDLR replacement University of Pennsylvania Completed/NCT00004809 [41,42]
HoFH ASO/ISIS 301012 ApoB inhibition Genzyme/ISIS Completed/NCT00607373 [58]
HetFH ASO/ISIS 301012 ApoB inhibition Genzyme/ISIS Completed/NCT00281008, NCT 00362180 [61,62]
HoFH siRNA/PRO-040201 ApoB inhibition Tekmira Completed/NCT00927459 [65,201]
LPL deficiency AAV1/AMT-011 LPL replacement Amsterdam Molecular Therapeutics Ongoing/NCT00891306 [145]

AAV: Adenoassociated viruses; ASO: Antisense oligonucleotide; HetFH: Heterozygous famial hypercholesterolemia; HoFH: Homozygous hypercholesterolemia; LDLR: LDL receptor; LPL: Lipoprotein lipase.