Research & Publications

Delivery of an adeno-associated virus vector into CSF attenuates central nervous system disease in mucopolysaccharidosis type II mice

Human Gene Therapy, August 10, 2016

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

Molecular Genetics and Metabolism, July 4, 2016

Intrathecal Gene Therapy Corrects CNS Pathology in a Feline Model of Mucopolysaccharidosis I
Molecular Therapy, July 16, 2014

Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I/II trial
Human Gene Therapy, May 5, 2014

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B
The New England Journal of Medicine, December 22, 2011

Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN
Nature Biotechnology, February 28, 2010