Stem Cell Therapies for the Lysosomal Storage Diseases – the Quintessential Neurodegenerative Diseases

Philip   Hitchins   Schwartz; David   Joseph   Brick

Abstract

As a novel neurotherapeutic strategy, stem cell transplantation has received considerable attention. However, little focus of this attention has been devoted to the probabilities of success of stem cell therapies for specific neurological disorders. Given the complexities of the cellular organization of the nervous system and the manner in which it is assembled during development, it seems unlikely that a cellular replacement strategy will succeed for any but the simplest of neurological disorders in the near future. A general strategy for stem cell transplantation to prevent or minimize neurological disorders is much more likely to succeed. The lysosomal storage diseases represent the quintessential neurodegenerative diseases for which preventative stem cell transplantation will both likely succeed and set the stage for therapeutic approaches to other neurodegenerative diseases.

Keywords: Stem cells, lysosomal storage diseases, stem cell transplantation, cellular therapy, nherited metabolic diseases

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