Search Result "Sandhoff disease"
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
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
Combined Therapies for Lysosomal Storage Diseases
Journal: Current Molecular Medicine
Volume: 15 Issue: 8 Year: 2015 Page: 746-771
Author(s): M. Gabig-CimiÅska,J. Jakóbkiewicz-Banecka,M. Malinowska,A. Kloska,E. Piotrowska,I. Chmielarz,M. Moskot,A. WÄgrzyn,G. WÄgrzyn
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
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
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
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
Clinical Utility of Neuronal Cells Directly Converted from Fibroblasts of Patients for Neuropsychiatric Disorders: Studies of Lysosomal Storage Diseases and Channelopathy
Journal: Current Molecular Medicine
Volume: 15 Issue: 2 Year: 2015 Page: 138-145
Author(s): S. Kano,M. Yuan,R.A. Cardarelli,G. Maegawa,N. Higurashi,M. Gaval-Cruz,A.M. Wilson,C. Tristan,M.A. Kondo,Y. Chen,M. Koga,C. Obie,K. Ishizuka,S. Seshadri,R. Srivastava,T.A. Kato,Y. Horiuchi,T.W. Sedlak,Y. Lee,J.L. Rapoport,S. Hirose,H. Okano,D. Valle,P. O'Donnell,A. Sawa,M. Kai