Novel treatment for neuronopathic lysosomal storage diseases--cell therapy/gene therapy

Most lysosomal storage diseases (LSD) exhibit neurological symptoms and there has been limited success in their treatment. Innovative treatments employing novel therapy or gene therapy may offer the prospect of improvement. Recent attempts to treat the neurological forms of LSD include neural stem cell therapy, mesenchymal stem cell therapy, hematopoietic stem cell therapy and gene therapy. Additional approaches have included substrate deprivation/chaperone therapy for the treatment of LSD. This article reviews these new technologies, discusses recent progress, and suggests their possible application.