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.

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 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 11/01/2016

Delivery of an Adeno-Associated Virus Vector into Cerebrospinal Fluid Attenuates Central Nervous System Disease in Mucopolysaccharidosis Type II Mice.

Hinderer C, Katz N, Louboutin JP, Bell P, Yu H, Nayal M, Kozarsky K, O'Brien WT, Goode T, Wilson JM

Human Gene Therapy

Publication 9/01/2016

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

Hinderer C, Bell P, Louboutin JP, Katz N, Zhu Y, Lin G, Choa R, Bagel J, O'Donnell P, Fitzgerald CA, Langan T, Wang P, Casal ML, Haskins ME, Wilson JM

Molecular Genetics And Metabolism