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
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
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