Genome Editing for Sickle Cell Disease: A Little BCL11A Goes a Long Way

Mol Ther. 2017 Mar 1;25(3):561-562. doi: 10.1016/j.ymthe.2017.02.003. Epub 2017 Feb 9.
No abstract available

Publication types

  • Editorial

MeSH terms

  • Anemia, Sickle Cell / genetics
  • Anemia, Sickle Cell / therapy*
  • CRISPR-Cas Systems
  • Carrier Proteins / genetics*
  • Gene Editing / methods*
  • Genetic Therapy / methods
  • Humans
  • Mutation
  • Nuclear Proteins / genetics*
  • Repressor Proteins
  • Zinc Finger Nucleases / metabolism

Substances

  • BCL11A protein, human
  • Carrier Proteins
  • Nuclear Proteins
  • Repressor Proteins
  • Zinc Finger Nucleases