Search Result "Tay-Sachs disease"


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

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

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

Induced Pluripotent Stem Cell Technology: A Paradigm Shift in Medical Science for Drug Screening and Disease Modeling

Journal: Current Medicinal Chemistry
Volume: 24 Issue: 39 Year: 2017 Page: 4368-4398
Author(s): Meera Nair,Sardul Singh Sandhu,Anil Kumar Sharma

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

Misfolded Proteins and Human Diseases

Journal: Protein & Peptide Letters
Volume: 11 Issue: 6 Year: 2004 Page: 593-600
Author(s): M. S. Nayeem, Rizwan H. Khan

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

Review Article

Global View on Rare Diseases: A Mini Review

Journal: Current Medicinal Chemistry
Volume: 24 Issue: 29 Year: 2017 Page: 3153-3158
Author(s): Blanka Klimova,Michael Storek,Martin Valis,Kamil Kuca

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

Somatic Genome Variations in Health and Disease

Journal: Current Genomics
Volume: 11 Issue: 6 Year: 2010 Page: 387-396
Author(s): I. Y. Iourov, S. G. Vorsanova, Y. B. Yurov

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