Publication
12/01/2016
Effects of Self-Complementarity, Codon Optimization, Transgene, and Dose on Liver Transduction with AAV8.
Bell P, Wang L, Chen SJ, Yu H, Zhu Y, Nayal M, He Z, White J, Lebel-Hagan D, Wilson JM
Human Gene Therapy Methods
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
3/01/2016
A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice.
Yang Y, Wang L, Bell P, McMenamin D, He Z, White J, Yu H, Xu C, Morizono H, Musunuru K, Batshaw ML, Wilson JM
Nature Biotechnology
Publication
2/01/2016
Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII.
Gurda BL, De Guilhem De Lataillade A, Bell P, Zhu Y, Yu H, Wang P, Bagel J, Vite CH, Sikora T, Hinderer C, Calcedo R, Yox AD, Steet RA, Ruane T, O'Donnell P, Gao G, Wilson JM, Casal M, Ponder KP, Haskins ME
Molecular Therapy : The Journal Of The American Society Of Gene Therapy
