Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial

Membrane-Associated Heparan Sulfate Proteoglycan Is a Receptor for Adeno-Associated Virus Type 2 Virions

Candace Summerford, R. Jude Samulski · Journal of Virology · 1998 · 1.4K citations · Full text

Life Sciences, Biology, Microbiology +17

AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial

Peter A. LeWitt, Ali R. Rezai, Maureen A. Leehey et al. · The Lancet Neurology · 2011 · 672 citations

Medicine, Neurology, Neuroscience +13

Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis by CNS Administration of a Serotype 2 Adeno-Associated Virus Expressing CLN2 cDNA

Stefan Worgall, Dolan Sondhi, Neil R. Hackett et al. · Human Gene Therapy · 2008 · 404 citations

Medicine, Neurology, Neuroscience +16

Long-Term Follow-Up After Gene Therapy for Canavan Disease

Paola Leone, David Shera, Scott McPhee et al. · Science Translational Medicine · 2012 · 325 citations · Full text

Medicine, Neurology, Neuroscience +14

Intracerebral Administration of Adeno-Associated Viral Vector Serotype rh.10 Carrying Human <i>SGSH</i> and <i>SUMF1</i> cDNAs in Children with Mucopolysaccharidosis Type IIIA Disease: Results of a Phase I/II Trial

Marc Tardieu, Michel Zérah, B. Husson et al. · Human Gene Therapy · 2014 · 270 citations

Medicine, Neurology, Pediatrics +17