Delivering Hematopoietic Stem Cell Gene Therapy Treatments for Neurological Lysosomal Diseases

ACS Chem Neurosci. 2019 Jan 16;10(1):18-20. doi: 10.1021/acschemneuro.8b00408. Epub 2018 Aug 23.


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.

Keywords: enzyme replacement therapy; gene therapy; hematopoietic stem cells; lysosomal storage diseases; mucopolysaccharidoses.

Publication types

  • Review

MeSH terms

  • Animals
  • Brain / physiology
  • Gene Transfer Techniques* / trends
  • Genetic Therapy / methods*
  • Genetic Therapy / trends
  • Hematopoietic Stem Cell Transplantation / methods*
  • Hematopoietic Stem Cell Transplantation / trends
  • Hematopoietic Stem Cells / physiology
  • Humans
  • Lysosomal Storage Diseases / genetics
  • Lysosomal Storage Diseases / therapy*
  • Nervous System Diseases / genetics
  • Nervous System Diseases / therapy*