Widespread correction of central nervous system disease after intracranial gene therapy in a feline model of Sandhoff disease

Global CNS Transduction of Adult Mice by Intravenously Delivered rAAVrh.8 and rAAVrh.10 and Nonhuman Primates by rAAVrh.10

(2014) Bin Yang et al.   MOLECULAR THERAPY

Sustained Normalization of Neurological Disease after Intracranial Gene Therapy in a Feline Model

(2014) V. J. McCurdy et al.   Science Translational Medicine

Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates

(2013) S J Gray et al.   GENE THERAPY

Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy

(2013) Virginia Haurigot et al.   JOURNAL OF CLINICAL INVESTIGATION

Therapeutic Response in Feline Sandhoff Disease Despite Immunity to Intracranial Gene Therapy

(2013) Allison M Bradbury et al.   MOLECULAR THERAPY

A lysosome-to-nucleus signalling mechanism senses and regulates the lysosome via mTOR and TFEB

(2012) Carmine Settembre et al.   EMBO JOURNAL

Gene Transfer Corrects Acute GM2 Gangliosidosis—Potential Therapeutic Contribution of Perivascular Enzyme Flow

(2012) M Begoña Cachón-González et al.   MOLECULAR THERAPY

Merits of Combination Cortical, Subcortical, and Cerebellar Injections for the Treatment of Niemann-Pick Disease Type A

(2012) Jie Bu et al.   MOLECULAR THERAPY

Therapeutic Effects of Stem Cells and Substrate Reduction in Juvenile Sandhoff Mice

(2012) J. R. Arthur et al.   NEUROCHEMICAL RESEARCH

Adeno-Associated Virus Serotype 9 Transduction in the Central Nervous System of Nonhuman Primates

(2011) Lluis Samaranch et al.   HUMAN GENE THERAPY

Therapeutic Potential of Intracerebroventricular Replacement of Modified Human β-Hexosaminidase B for GM2 Gangliosidosis

(2011) Kazuhiko Matsuoka et al.   MOLECULAR THERAPY

Highly phosphomannosylated enzyme replacement therapy for GM2 gangliosidosis

(2010) Daisuke Tsuji et al.   ANNALS OF NEUROLOGY

Bone marrow transplantation in canine GM1 gangliosidosis

(2010) John S. O'Brien et al.   CLINICAL GENETICS

Early versus late treatment of spinal cord compression with long-term intrathecal enzyme replacement therapy in canine mucopolysaccharidosis type I

(2010) Patricia I. Dickson et al.   MOLECULAR GENETICS AND METABOLISM

An open-label Phase I/II clinical trial of pyrimethamine for the treatment of patients affected with chronic GM2 gangliosidosis (Tay–Sachs or Sandhoff variants)

(2010) Joe T.R. Clarke et al.   MOLECULAR GENETICS AND METABOLISM

Pyrimethamine increases β-hexosaminidase A activity in patients with Late Onset Tay Sachs

(2010) Etty Osher et al.   MOLECULAR GENETICS AND METABOLISM

Safe, Efficient, and Reproducible Gene Therapy of the Brain in the Dog Models of Sanfilippo and Hurler Syndromes

(2010) N Matthew Ellinwood et al.   MOLECULAR THERAPY

AAV-Mediated Gene Delivery in Adult GM1-Gangliosidosis Mice Corrects Lysosomal Storage in CNS and Improves Survival

(2010) Rena C. Baek et al.   PLoS One

Miglustat in late-onset Tay-Sachs disease: a 12-month, randomized, controlled clinical study with 24 months of extended treatment

(2009) Barbara E Shapiro et al.   GENETICS IN MEDICINE

Human α-Iduronidase Gene Transfer Mediated by Adeno-Associated Virus Types 1, 2, and 5 in the Brain of Nonhuman Primates: Vector Diffusion and Biodistribution

(2009) Carine Ciron et al.   HUMAN GENE THERAPY

Substrate reduction therapy in juvenile GM2 gangliosidosis

(2009) Gustavo H.B. Maegawa et al.   MOLECULAR GENETICS AND METABOLISM

Systematic Evaluation of AAV Vectors for Liver directed Gene Transfer in Murine Models

(2009) Lili Wang et al.   MOLECULAR THERAPY

Mechanisms of distribution of mouse β-galactosidase in the adult GM1-gangliosidosis brain

(2008) M L D Broekman et al.   GENE THERAPY

Comparative Analysis of Brain Lipids in Mice, Cats, and Humans with Sandhoff Disease

(2008) Rena C. Baek et al.   LIPIDS