Therapeutic benefit after intracranial gene therapy delivered during the symptomatic stage in a feline model of Sandhoff disease

AAVrh10 Gene Therapy Ameliorates Central and Peripheral Nervous System Disease in Canine Globoid Cell Leukodystrophy (Krabbe Disease)

(2018) Allison M. Bradbury et al.   HUMAN GENE THERAPY

Direct Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain

(2017) Diane Golebiowski et al.   HUMAN GENE THERAPY

AAV-mediated gene delivery attenuates neuroinflammation in feline Sandhoff disease

(2017) Allison M. Bradbury et al.   NEUROSCIENCE

Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII

(2016) Brittney L Gurda et al.   MOLECULAR THERAPY

Biomarkers for disease progression and AAV therapeutic efficacy in feline Sandhoff disease

(2015) Allison M. Bradbury et al.   EXPERIMENTAL NEUROLOGY

Mucopolysaccharidosis-like phenotype in feline Sandhoff disease and partial correction after AAV gene therapy

(2015) Heather L. Gray-Edwards et al.   MOLECULAR GENETICS AND METABOLISM

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

(2014) V J McCurdy et al.   GENE THERAPY

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

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

Reversibility of neuropathology in Tay–Sachs-related diseases

(2013) María-Begoña Cachón-González et al.   HUMAN MOLECULAR GENETICS

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 Single Intravenous rAAV Injection as Late as P20 Achieves Efficacious and Sustained CNS Gene Therapy in Canavan Mice

(2013) Seemin Seher Ahmed et al.   MOLECULAR THERAPY

Tay Sachs disease in Australia: reduced disease incidence despite stable carrier frequency in Australian Jews

(2012) Raelia M Lew et al.   MEDICAL JOURNAL OF AUSTRALIA

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

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

GM2 gangliosidosis in a UK study of children with progressive neurodegeneration: 73 cases reviewed

(2011) NICHOLAS J SMITH et al.   DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY

Natural History of Infantile GM2 Gangliosidosis

(2011) A. E. Bley et al.   PEDIATRICS

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

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

Impaired clearance of accumulated lysosomal glycogen in advanced Pompe disease despite high-level vector-mediated transgene expression

(2009) Baodong Sun et al.   JOURNAL OF GENE MEDICINE

Miglustat therapy in juvenile Sandhoff disease

(2009) C. M. E. Tallaksen et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Substrate reduction therapy in juvenile GM2 gangliosidosis

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

Neurodegenerative lysosomal storage disease in European Burmese cats with hexosaminidase β-subunit deficiency

(2009) Allison M. Bradbury et al.   MOLECULAR GENETICS AND METABOLISM

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