Research & Publications

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.

Supplementary Data

Kassim et al. Human Gene Therapy, January 2013

Gene Therapy in a Humanized Mouse Model of Familial Hypercholesterolemia Leads to Marked Regression of Atherosclerosis.

Kassim et al. PLoS One, October 2010

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

Supplementary Data

Greig et al. Human Gene Therapy, March 2017

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

Supplementary Data

Greig et al. Human Gene Therapy, March 2017

Delivery of an adeno-associated virus vector into CSF attenuates central nervous system disease in mucopolysaccharidosis type II mice

Human Gene Therapy, August 10, 2016

Neonatal tolerance induction enables accurate evaluation of gene therapy for MPS I in a canine model

Molecular Genetics and Metabolism, July 4, 2016

Intrathecal Gene Therapy Corrects CNS Pathology in a Feline Model of Mucopolysaccharidosis I
Molecular Therapy, July 16, 2014

Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I/II trial
Human Gene Therapy, May 5, 2014

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B
The New England Journal of Medicine, December 22, 2011

Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN
Nature Biotechnology, February 28, 2010