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Hematopoietic stem cell collection for sickle cell disease gene therapy.

Alexis LeonardMitchell J Weiss
Published in: Current opinion in hematology (2024)
Here we review the current knowledge on HSC collection from individuals with SCD and potential improvements that may enhance the safety, efficacy, and availability of gene therapy for this disorder.
Keyphrases
  • sickle cell disease
  • gene therapy
  • hematopoietic stem cell
  • healthcare
  • copy number
  • genome wide
  • genome wide identification
  • human health
  • risk assessment
  • dna methylation
  • transcription factor
  • genome wide analysis