Gene Therapy for Pediatric Neurologic Disease

Abstract

Pediatric lysosomal and peroxisomal storage disorders, leukodystrophies, and motor neuron diseases can have devastating neurologic manifestations. Despite efforts to exploit cross-correction to treat these monogenic disorders for several decades, definitive treatment has yet to be identified. This review explores recent attempts to transduce autologous hematopoietic stem cells with functional gene or provide therapeutic gene in vivo. Specifically, we discuss the rationale behind efforts to treat pediatric neurologic disorders with gene therapy, outline the specific disorders that have been targeted at this time, and review recent and current clinical investigations with attention to the future direction of therapy efforts.

Keywords: Ex vivo; Gene therapy; In vivo; Leukodystrophies; Lysosomal storage disorders; Motor neuron disease; Neurologic disorders; Peroxisomal storage disorders.