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
