Article

CD117 epitope-shielded hematopoietic stem cell transplantation with toxin-free conditioning and in vivo selection ameliorates a β-thalassemia model

2026-07-08

Abstract excerpt

<h4>SUMMARY</h4> Clinical evidence demonstrates that ex vivo gene therapy and genome engineering of hematopoietic stem and progenitor cells (HSPCs) could represent one-time cures. However, while genome editing itself has become increasingly efficient and precise, the toxic conditioning required for hematopoietic stem cell transplantation remains a major barrier to broad clinical implementation of these otherwise...

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Literature Corpus work: 343177d8-17b5-5b32-95e1-709c9399f11f
DOI: 10.64898/2026.07.07.736903

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