Search Result "Sandhoff disease"


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

Molecular Pathologies of and Enzyme Replacement Therapies for Lysosomal Diseases

Journal: CNS & Neurological Disorders - Drug Targets
Volume: 5 Issue: 4 Year: 2006 Page: 401-413
Author(s): Hitoshi Sakuraba, Makoto Sawada, Fumiko Matsuzawa, Sei-ichi Aikawa, Yasunori Chiba, Yoshifumi Jigami, Kohji Itoh

Review Article

Neural Stem Cell Transplantation and CNS Diseases

Journal: CNS & Neurological Disorders - Drug Targets
Volume: 15 Issue: 8 Year: 2016 Page: 881-886
Author(s): Rodolfo Gonzalez, Milton H. Hamblin, Jean-Pyo Lee

Review Article open access plus

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

Combined Therapies for Lysosomal Storage Diseases

Journal: Current Molecular Medicine
Volume: 15 Issue: 8 Year: 2015 Page: 746-771
Author(s): M. Gabig-Cimińska,J. Jakóbkiewicz-Banecka,M. Malinowska,A. Kloska,E. Piotrowska,I. Chmielarz,M. Moskot,A. Węgrzyn,G. Węgrzyn

Treatment of Lysosomal Storage Diseases: Recent Patents and Future Strategies

Journal: Recent Patents on Endocrine, Metabolic & Immune Drug Discovery
Volume: 8 Issue: 1 Year: 2014 Page: 9-25
Author(s): Saida Ortolano,Irene Vieitez,Carmen Navarro,Carlos Spuch

Epigenetic and Disease Targets by Polyphenols

Journal: Current Pharmaceutical Design
Volume: 19 Issue: 3 Year: 2013 Page: 6156-6185
Author(s): Min-Hsiung Pan, Ching-Shu Lai, Jia-Ching Wu, Chi-Tang Ho

Lysosomal Storage Diseases and the Blood-Brain Barrier

Journal: Current Pharmaceutical Design
Volume: 14 Issue: 1 Year: 2008 Page: 1566-1580
Author(s): David J. Begley, Charles C. Pontikis, Maurizio Scarpa

Fabry Disease Cardiomyopathy: from Genes to Clinical Manifestations

Journal: Current Pharmaceutical Biotechnology
Volume: 13 Issue: 1 Year: 2012 Page: 2477-2484
Author(s): Attila Bela Kertesz, Istvan Edes

Recent Developments in Therapeutic Approaches for Lysosomal Storage Diseases

Journal: Recent Patents on CNS Drug Discovery (Discontinued)
Volume: 6 Issue: 1 Year: 2011 Page: 1-19
Author(s): Lorena Urbanelli, Alessandro Magini, Alice Polchi, Mario Polidoro, Carla Emiliani

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