Hematopoietic Gene Therapies for Metabolic and Neurologic Diseases

Hematol Oncol Clin North Am. 2017 Oct;31(5):869-881. doi: 10.1016/j.hoc.2017.06.004. Epub 2017 Jul 23.


Increasingly, patients affected by metabolic diseases affecting the central nervous system and neuroinflammatory disorders receive hematopoietic cell transplantation (HCT) in the attempt to slow the course of their disease, delay or attenuate symptoms, and improve pathologic findings. The possible replacement of brain-resident myeloid cells by the transplanted cell progeny contributes to clinical benefit. Genetic engineering of the cells to be transplanted (hematopoietic stem cell) may endow the brain myeloid progeny of these cells with enhanced or novel functions, contributing to therapeutic effects.

Keywords: Degeneration; Gene therapy; Hematopoietic stem cells; Nervous system; Storage.

Publication types

  • Review

MeSH terms

  • Animals
  • Gene Expression
  • Genetic Engineering
  • Genetic Therapy* / adverse effects
  • Genetic Therapy* / methods
  • Genetic Vectors / genetics
  • Hematopoietic Stem Cell Transplantation
  • Hematopoietic Stem Cells / cytology
  • Hematopoietic Stem Cells / metabolism*
  • Humans
  • Metabolic Diseases / genetics*
  • Metabolic Diseases / therapy*
  • Microglia / metabolism
  • Nervous System Diseases / genetics*
  • Nervous System Diseases / therapy*
  • Transduction, Genetic
  • Transgenes