Mucopolysaccharidosis Type I Phenotypically Corrected with Edited Hematopoietic Stem Cells: Instead of altering the IDUA gene, a protein was inserted in a repurposable place in the genome known as a "safe harbor locus"

Am J Med Genet A. 2020 Feb;182(2):275-276. doi: 10.1002/ajmg.a.61223.
No abstract available

MeSH terms

  • Animals
  • Antigens, CD34 / genetics
  • CRISPR-Cas Systems / genetics
  • Gene Editing / methods
  • Hematopoietic Stem Cell Transplantation*
  • Hematopoietic Stem Cells / metabolism
  • Humans
  • Iduronidase / genetics*
  • Mice
  • Mucopolysaccharidosis I / genetics*
  • Mucopolysaccharidosis I / pathology
  • Mucopolysaccharidosis I / therapy
  • Phenotype
  • Receptors, CCR5 / genetics*

Substances

  • Antigens, CD34
  • CCR5 protein, human
  • Receptors, CCR5
  • IDUA protein, human
  • Iduronidase