Synapse
⌘+K
Synapse
PulseExploreClubsResearchersJournals
Instagram
HomeClubsExplore
December 8, 2025BloodOpen Access

Multiplex base editing enhances fetal hemoglobin production in sickle cell disease erythroid cells

View Full Paper
Ask AI
Bookmark
Share

Authors

KJKun JiaUniversity of California, San FranciscoESEric SoupèneUniversity of California, San FranciscoMCMarco CorderoUniversity of California, San Francisco

Discussion

Loading...

Member takes

Implication

Observational analysis improved fetal hemoglobin levels in sickle cell disease cells, suggesting multiplex editing strategies enhance red blood cell production.

Key Points

  • Fetal hemoglobin levels increased by 84.6% compared to 7.9% in unedited cells, indicating enhanced therapeutic benefits.
  • Human hematopoietic stem and progenitor cells were edited using multiplex strategies, achieving up to 87% allele editing efficiency without compromising viability.
  • No signs of genotoxicity were observed in the edited human hematopoietic stem and progenitor cells, ensuring safety of the approach.
  • These findings propose a new framework for improving gene editing outcomes in sickle cell disease through combinatorial strategies.

Cite This Study

Jia et al. (2025) studied this question.

synapsesocial.com/papers/69362f3d4fa91c937236d48fhttps://doi.org/10.1182/blood-2025-2531
View Full Paper
Ask AI
Bookmark
Share

Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Toward ‘n of one’ gene therapies: Scalable ex vivo adenine base editing via lipid nanoparticles in a sickle cell disease model2025 · 1 citations
  2. 2Targeted disruption of BCL11A ZnF4 enhances fetal hemoglobin in β-thalassemia and sickle cell disease: A druggable approach2025
  3. 3Base editing of β 0 thalassemia mutations as a therapeutic strategy for β-hemoglobinopathies: efficacy and genotoxicity studies2025
  4. 4Engraftment and persistence of HBB base-edited hematopoietic stem cells in nonhuman primates2025 · 6 citations
  5. 5In Vivo correction of the sickle cell disease mutation in hematopoietic stem cells using RNA gene writers2025 · 2 citations