Combinatorial base editing couples disease correction with lineage amplification in hematopoietic stem and progenitor cells
, establishing a modular strategy that pairs disease correction with lineage amplification to improve therapeutic potency.
Authors
- Elisabetta Manuela Foppiani (ORCID: https://orcid.org/0000-0003-1595-8759)
- Mark C. Walters (ORCID: https://orcid.org/0000-0002-6515-4559)
- Vivien Sheehan (ORCID: https://orcid.org/0000-0003-2837-2255)
- M. Kyle Cromer (ORCID: https://orcid.org/0000-0002-8198-5010)
- Devesh Sharma (ORCID: https://orcid.org/0000-0002-3801-7276)
- Roshani Sinha (ORCID: https://orcid.org/0000-0001-6971-2725)
- Tippi C. MacKenzie (ORCID: https://orcid.org/0000-0001-5232-809X)
- Eric Soupène (ORCID: https://orcid.org/0000-0002-1965-5117)
- Benjamin J. Lesch (ORCID: https://orcid.org/0000-0002-2511-3517)
- Marco Cordero
- Rachel Choi (ORCID: https://orcid.org/0009-0001-6075-8687)
- Simon N. Chu (ORCID: https://orcid.org/0000-0003-3863-1548)
- Michael A. Pendergast (ORCID: https://orcid.org/0009-0000-3560-0290)
- Zachary Kostamo (ORCID: https://orcid.org/0009-0007-5571-8713)
- Xinyi Zhang
- Xinjie Yu
Institutions
- Emory University (US)
- University of California, San Francisco (US)
- UCSF Benioff Children's Hospital (US)
- Children's Healthcare of Atlanta (US)
- Broad Center (US)
Publication Details
- Journal
- bioRxiv (Cold Spring Harbor Laboratory)
- Published
- 2026-04-14
- DOI
- https://doi.org/10.64898/2026.04.13.718029
- Primary Topic
- CRISPR and Genetic Engineering
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- National Science Foundation Graduate Research Fellowship Program