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In utero HSC transplantation for sickle cell disease: A potential therapeutic approach that overcomes complications of current therapies

Babatunde OO, Bibby MG, Atala A, Almeida-Porada G, Porada CD (2026) In utero HSC transplantation for sickle cell disease: A potential therapeutic approach that overcomes complications of current therapies. Prenat Diagn doi: 10.1002/pd.70142 PMID: 41936060

Objective: To examine current evidence and recent advances for treatment of sickle cell disease (SCD).

Summary: Biotinylated anti‐c‐kit/CD117 mAb coupled to a streptavidin‐conjugated saporin has been used to selectively deplete host HSC while preserving the host’s immune system. A single intravenous dose of the anti‐CD45‐saporin ADC enabled > 90% donor (congenic) hematopoietic engraftment and full correction of the SCD phenotype.

Related Products: Anti-CD117-SAP (Cat. #IT-83), Streptavidin-ZAP (Cat. #IT-27), Anti-CD45.2-SAP (Cat. #IT-91)

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