Intracisternal cyclodextrin prevents cerebellar dysfunction and Purkinje cell death in feline Niemann-Pick type C1 disease

Charles H. Vite, Jessica Bagel, Gary P. Swain, +16

Science Translational Medicine

Evaluation of Intrathecal Routes of Administration for Adeno-Associated Viral Vectors in Large Animals

Christian Hinderer, Peter Bell, Nathan Katz, +12

Human Gene Therapy

ImmunologyTransduction EfficiencyGene DeliveryBiomedical EngineeringIntrathecal Routes +17

Intrathecal Gene Therapy Corrects CNS Pathology in a Feline Model of Mucopolysaccharidosis I

Christian Hinderer, Peter Bell, Brittney L. Gurda, +10

Molecular Therapy

Identification of Niemann-Pick C1 disease biomarkers through sphingolipid profiling

Martin Fan, Rohini Sidhu, Hideji Fujiwara, +10

Journal of Lipid Research

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

Allison M. Bradbury, Mohammed Rafi, Jessica Bagel, +13

Human Gene Therapy

ImmunologyPathologyCentral Nervous SystemsDisease CorrectionExperimental Neuropathology +16

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

Brittney L. Gurda, Adrien de Guilhem de Lataillade, Peter Bell, +17

Molecular Therapy

Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates

Christian Hinderer, Peter Bell, Jean-Pierre Louboutin, +14

Molecular Therapy

Krabbe disease successfully treated via monotherapy of intrathecal gene therapy

Allison M. Bradbury, Jessica Bagel, Duc Nguyen, +16

Journal of Clinical Investigation

Cell TherapyIn Vivo Gene TherapyImmunologyPathologyCell Replacement Therapy +18

Cholesterol homeostatic responses provide biomarkers for monitoring treatment for the neurodegenerative disease Niemann–Pick C1 (NPC1)

Brett A. Tortelli, Hideji Fujiwara, Jessica Bagel, +12

Human Molecular Genetics

Neurochemical BiomarkersSocial SciencesNeurobiology Of DiseaseNpc1 DiseaseNeurology +16

Efficacy and Safety of a Krabbe Disease Gene Therapy

Juliette Hordeaux, Brianne A Jeffrey, Jinlong Jian, +10

Human Gene Therapy