Search Result "GM2 gangliosidosis"


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Genetics and Therapies for GM2 Gangliosidosis

Journal: Current Gene Therapy
Volume: 18 Issue: 2 Year: 2018 Page: 68-89
Author(s): Maria Begona Cachon-Gonzalez,Eva Zaccariotto,Timothy Martin Cox

Research Article

Treatment of GM2 Gangliosidosis in Adult Sandhoff Mice Using an IntravenousSelf-Complementary Hexosaminidase Vector

Journal: Current Gene Therapy
Volume: 22 Issue: 3 Year: 2022 Page: 262-276
Author(s): Karlaina JL. Osmon,Patrick Thompson,Evan Woodley,Subha Karumuthil-Melethil,Cliff Heindel,John G. Keimel,William F. Kaemmerer,Steven J. Gray,Jagdeep S. Walia

Research Article

AAV9-coGLB1 Improves Lysosomal Storage and Rescues Central NervousSystem Inflammation in a Mutant Mouse Model of GM1 Gangliosidosis

Journal: Current Gene Therapy
Volume: 22 Issue: 4 Year: 2022 Page: 352-365
Author(s):

Miglustat: Substrate Reduction Therapy for Lysosomal Storage Disorders Associated with Primary Central Nervous System Involvement

Journal: Recent Patents on CNS Drug Discovery (Discontinued)
Volume: 1 Issue: 1 Year: 2006 Page: 77-82
Author(s): Gregory M. Pastores

GABAergic Pharmacotherapy in the Treatment of Motor Disorders of the Central Nervous System

Journal: Current Pharmaceutical Design
Volume: 21 Issue: 34 Year: 2015 Page: 4989-4995
Author(s): Jose Gazulla,Carlota Ruiz-Gazulla,Maria Tintore

Review Article

New Advanced Strategies for the Treatment of Lysosomal Diseases Affecting the Central Nervous System

Journal: Current Pharmaceutical Design
Volume: 25 Issue: 1 Year: 2019 Page: 1933-1950
Author(s): Maria R. Gigliobianco,Piera Di Martino,Siyuan Deng,Cristina Casadidio,Roberta Censi

Review Article

Structure-to-function Relationship of Carbohydrates in the Mechanism of Lysosomal Storage Disorders (LSDs)

Journal: Current Organic Chemistry
Volume: 21 Issue: 2 Year: 2017 Page: 2719-2730
Author(s): Mirela Sarbu,Claudia Cozma,Alina D. Zamfir

Mini-Review Article

Clinical Development of Cell Therapies to Halt Lysosomal Storage Diseases:Results and Lessons Learned

Journal: Current Gene Therapy
Volume: 22 Issue: 3 Year: 2022 Page: 191-213
Author(s): Valeria Graceffa

The Two Faces of Iminoalditols: Powerful Inhibitors Trigger Glycosidase Activation

Journal: Current Enzyme Inhibition
Volume: 8 Issue: 1 Year: 2012 Page: 47-99
Author(s): Tanja M. Wrodnigg, Arnold E. Stutz

Journal: Current Stem Cell Research & Therapy
Volume: 3 Issue: 2 Year: 2008 Page: 88-98
Author(s): Philip Hitchins Schwartz, David Joseph Brick

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