Delivering Hematopoietic Stem Cell Gene Therapy Treatments for Neurological Lysosomal Diseases.

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Abstract

Neurological lysosomal storage diseases are rare, inherited conditions resulting mainly from lysosomal enzyme deficiencies. Current treatments, such as enzyme replacement therapy and hematopoietic stem cell transplantation, fail to effectively treat neurological disease due to insufficient brain delivery of the missing enzyme. Ex vivo gene therapy approaches to overexpress the missing enzyme in hematopoietic stem cells prior to transplant are an emerging technology that has the potential to offer a viable therapy for patients with these debilitating diseases.
Original languageEnglish
JournalACS Chemical Neuroscience
Early online date23 Aug 2018
DOIs
Publication statusPublished - 2018

Keywords

  • mucopolysaccharidoses
  • enzyme replacement therapy
  • gene therapy
  • hematopoietic stem cells
  • Lysosomal storage diseases

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