1020 Locust Street
Suite 393
Philadelphia, PA 19107
(215) 503-5715
Suite 393
Philadelphia, PA 19107
(215) 503-5715
Most Recent Peer-reviewed Publications
- Extended normal life after AAVrh10-mediated gene therapy in the mouse model of krabbe disease
- Effects of treatments on inflammatory and apoptotic markers in the CNS of mice with globoid cell leukodystrophy
- Insulin-like growth factor-1 provides protection against psychosine-induced apoptosis in cultured mouse oligodendrocyte progenitor cells using primarily the PI3K/Akt pathway
- Biochemical and pathological evaluation of long-lived mice with globoid cell leukodystrophy after bone marrow transplantation
- AAV-Mediated expression of galactocerebrosidase in brain results in attenuated symptoms and extended life span in murine models of globoid cell leukodystrophy
- Generation of transgenic mice expressing insulin-like growth factor-1 under the control of the myelin basic protein promoter: Increased myelination and potential for studies on the effects of increased IGF-1 on experimentally and genetically induced demyelination
- Generation of a mouse with low galactocerebrosidase activity by gene targeting: A new model of globoid cell leukodystrophy (Krabbe disease)
- Krabbe disease: Genetic aspects and progress toward therapy
- Evidence of diffuse brain pathololgy and unspecific genetic characterization in a patient with an atypical form of adult-onset Krabbe disease
- Globoid cell leukodystrophy in cairn and West Highland white terriers
- Genetic Galactocerebrosidase Deficiency (Globoid Cell Leukodystrophy, Krabbe Disease) in Rhesus Monkeys (Macaca mulatta)
- Analysis of the 5' flanking region of the human galactocerebrosidase (GALC) gene
- Molecular genetics of krabbe disease (Globoid cell Leukodystrophy): Diagnostic and clinical implications
- Prevalent mutations in the GALC gene of patients with Krabbe disease of Dutch and other European origin
- Characterization of the rhesus monkey galactocerebrosidase (GALC) cDNA and gene and identification of the mutation causing globoid cell leukodystrophy (Krabbe disease) in this primate
- Retroviral vector-mediated transfer of the galactocerebrosidase (GALC) cDNA leads to overexpression and transfer of GALC activity to neighboring cells
- Multiple mutations in the GALC gene in a patient with adult-onset Krabbe disease
- Two different mutations are responsible for Krabbe disease in the Druze and Moslem Arab populations in Israel
- Glucocorticoid responsiveness conferred by a cloned DNA binding protein.
- DNA binding proteins that amplify surfactant protein B gene expression: Isolation and characterization
