We invite you to discover Human Gene Therapy Clinical Development, a peer-reviewed journal publishing preclinical research and clinical trial data for gene and cell therapies.

Filter by author:

Publication 3/01/2018

Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN.

Hinderer C, Katz N, Buza EL, Dyer C, Goode T, Bell P, Richman LK, Wilson JM

Human Gene Therapy

Publication 2/16/2018

AAV8 Gene Therapy Rescues the Newborn Phenotype of a Mouse Model of Crigler-Najjar.

Greig JA, Nordin JML, Draper C, Bell P, Wilson JM

Human Gene Therapy

Publication 1/01/2018

Evaluation of Intrathecal Routes of Administration for Adeno-Associated Viral Vectors in Large Animals.

Hinderer C, Bell P, Katz N, Vite CH, Louboutin JP, Bote E, Yu H, Zhu Y, Casal ML, Bagel J, O'Donnell P, Wang P, Haskins ME, Goode T, Wilson JM

Human Gene Therapy

Publication 10/01/2017

Abnormal polyamine metabolism is unique to the neuropathic forms of MPS: potential for biomarker development and insight into pathogenesis.

Hinderer C, Katz N, Louboutin JP, Bell P, Tolar J, Orchard PJ, Lund TC, Nayal M, Weng L, Mesaros C, de Souza CFM, Dalla Corte A, Giugliani R, Wilson JM

Human Molecular Genetics

Publication 4/01/2017

AAV gene therapy corrects OTC deficiency and prevents liver fibrosis in aged OTC-knock out heterozygous mice.

Wang L, Bell P, Morizono H, He Z, Pumbo E, Yu H, White J, Batshaw ML, Wilson JM

Molecular Genetics And Metabolism

Publication 3/01/2017

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

Greig JA, Limberis MP, Bell P, Chen SJ, Calcedo R, Rader DJ, Wilson JM

Human Gene Therapy. Clinical Development