Short-hairpin RNA against aberrant HBBIVSI-110(G>A) mRNA restores β-globin levels in a novel cell model and acts as mono- and combination therapy for β-thalassemia in primary hematopoietic stem cells

Haematologica. 2018 Sep;103(9):e419-e423. doi: 10.3324/haematol.2018.189357. Epub 2018 Apr 26.
No abstract available

Publication types

  • Letter
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Alleles
  • Animals
  • Cell Line
  • Combined Modality Therapy
  • Gene Expression Regulation*
  • Hematopoietic Stem Cells / metabolism*
  • Humans
  • Mice
  • Mutation
  • RNA Interference
  • RNA, Messenger
  • RNA, Small Interfering / genetics*
  • beta-Globins / genetics*
  • beta-Thalassemia / genetics*
  • beta-Thalassemia / therapy*

Substances

  • RNA, Messenger
  • RNA, Small Interfering
  • beta-Globins