Gene Therapy for Gaucher's and Fabry Disease Using Viruses and Blood-Forming Cells
Retroviral-Mediated Transfer and Expression of Glucocerebrosidase and Ceramidtrihexosidase (a-Galactosidase A) cDNA's in Human Hematopoietic Progenitor Cells Retroviral-Mediated Transfer and Expression of Glucocerebrosidaseand Ceramidtrihexosidase (a-Galacto ...
Gaucher's Disease
Genetic: humanglucocerebrosidasecDNA
National Institute of Neurological Disorders and Stroke (NINDS)