Krabbe Disease: Prospects of Finding a Cure Using AAV Gene Therapy
Krabbe Disease (KD) is an autosomal metabolic disorder that affects both the central and peripheral nervous systems.It is caused by a OEM Screen Interface functional deficiency of the lysosomal enzyme, galactocerebrosidase (GALC), resulting in an accumulation of the toxic metabolite, psychosine.Psychosine accumulation affects many different cellula