Mohammad A Rafi Selected Research

Galactosylceramidase

8/2021	Advances in the Diagnosis and Treatment of Krabbe Disease.
1/2021	Can early treatment of twitcher mice with high dose AAVrh10-GALC eliminate the need for BMT?
1/2020	Conditions for combining gene therapy with bone marrow transplantation in murine Krabbe disease.
11/2015	Long-term Improvements in Lifespan and Pathology in CNS and PNS After BMT Plus One Intravenous Injection of AAVrh10-GALC in Twitcher Mice.
3/2015	Intravenous injection of AAVrh10-GALC after the neonatal period in twitcher mice results in significant expression in the central and peripheral nervous systems and improvement of clinical features.
11/2012	Extended normal life after AAVrh10-mediated gene therapy in the mouse model of Krabbe disease.
12/2009	Effects of treatments on inflammatory and apoptotic markers in the CNS of mice with globoid cell leukodystrophy.
5/2009	Significant correction of pathology in brains of twitcher mice following injection of genetically modified mouse neural progenitor cells.
11/2005	Insulin-like growth factor-1 provides protection against psychosine-induced apoptosis in cultured mouse oligodendrocyte progenitor cells using primarily the PI3K/Akt pathway.
9/2005	AAV2/5 vector expressing galactocerebrosidase ameliorates CNS disease in the murine model of globoid-cell leukodystrophy more efficiently than AAV2.

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Mohammad A Rafi Research Topics

Disease

12	Globoid Cell Leukodystrophy (Krabbe Disease) 08/2021 - 05/2005
3	Mucopolysaccharidoses 01/2021 - 01/2018
3	Neurodegenerative Diseases (Neurodegenerative Disease) 11/2012 - 05/2005
2	Neoplasms (Cancer) 08/2020 - 01/2015
2	Metachromatic Leukodystrophy (Sulfatide Lipidosis) 11/2013 - 06/2003
1	Neuroinflammatory Diseases 01/2022
1	Inflammation (Inflammations) 01/2021
1	Alzheimer Disease (Alzheimer's Disease) 01/2020
1	Brain Neoplasms (Brain Tumor) 01/2019
1	Brain Diseases (Brain Disorder) 01/2016
1	Lysosomal Storage Diseases (Lysosomal Storage Disease) 01/2013
1	Inborn Genetic Diseases (Disease, Hereditary) 01/2011
1	Central Nervous System Diseases (CNS Diseases) 09/2005
1	Demyelinating Diseases (Demyelinating Disease) 05/2004

Drug/Important Bio-Agent (IBA)

12	GalactosylceramidaseIBA 08/2021 - 05/2005
10	EnzymesIBA 01/2022 - 09/2005
3	Pharmaceutical PreparationsIBA 08/2020 - 01/2016
2	Cerebroside-Sulfatase (Arylsulfatase A)IBA 11/2013 - 06/2003
1	Amyloid beta-PeptidesIBA 01/2020
1	tau Proteins (tau Protein)IBA 01/2020
1	GlycosaminoglycansIBA 09/2019
1	Peptides (Polypeptides)IBA 01/2019
1	Biological ProductsIBA 01/2016
1	Proteins (Proteins, Gene)FDA Link 01/2013
1	Psychosine (Galactosylsphingosine)IBA 11/2005
1	Insulin (Novolin)FDA Link 05/2004
1	Insulin-Like Growth Factor I (IGF-1)IBA 05/2004
1	Myelin Basic ProteinIBA 05/2004
1	SulfatasesIBA 06/2003
1	Sulfoglycosphingolipids (Sulfatides)IBA 06/2003

Therapy/Procedure

6	Therapeutics 01/2021 - 01/2011
2	Drug Therapy (Chemotherapy) 08/2020 - 01/2016
2	Bone Marrow Transplantation (Transplantation, Bone Marrow) 01/2020 - 09/2005
1	Immunotherapy 01/2015
1	Hematopoietic Stem Cell Transplantation 05/2005