{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"institution":[{"name":"bioRxiv"}],"indexed":{"date-parts":[[2026,7,30]],"date-time":"2026-07-30T13:04:36Z","timestamp":1785416676496,"version":"3.56.0"},"posted":{"date-parts":[[2026,7,8]]},"group-title":"Bioengineering","reference-count":70,"publisher":"openRxiv","license":[{"start":{"date-parts":[[2026,7,8]],"date-time":"2026-07-08T00:00:00Z","timestamp":1783468800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100010663","name":"H2020 European Research council","doi-asserted-by":"crossref","award":["818806"],"award-info":[{"award-number":["818806"]}],"id":[{"id":"10.13039\/100010663","id-type":"DOI","asserted-by":"crossref"}]},{"id":[{"id":"https:\/\/ror.org\/02s6k3f65","id-type":"ROR","asserted-by":"publisher"}]},{"name":"US NIH","award":["R35GM118062"],"award-info":[{"award-number":["R35GM118062"]}]},{"name":"US NIH","award":["RM1HG009490"],"award-info":[{"award-number":["RM1HG009490"]}]},{"DOI":"10.13039\/100000011","name":"Howard Hughes Medical Institute","doi-asserted-by":"crossref","id":[{"id":"10.13039\/100000011","id-type":"DOI","asserted-by":"crossref"}]},{"DOI":"10.13039\/501100002347","name":"Bundesministerium f\u00fcr Bildung und Forschung","doi-asserted-by":"crossref","award":["EkoEstMed\u2013FKZ 01ZZ2015"],"award-info":[{"award-number":["EkoEstMed\u2013FKZ 01ZZ2015"]}],"id":[{"id":"10.13039\/501100002347","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"accepted":{"date-parts":[[2026,7,8]]},"abstract":"<jats:title>SUMMARY<\/jats:title>\n                <jats:p>\n                  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 curative therapies. In particular, the use of busulfan for myeloablative conditioning constitutes a major safety concern. While preclinical studies established CD117 as a promising target for antigen-specific therapy, clinical translation faced setbacks balancing efficacy and safety. To overcome current limitations, we generated a new CD117-blocking monoclonal antibody (CIM058) and demonstrate its potency to block wild-type HSPCs. To enable long-term blockade of host HSPCs even after transplantation, we used prime editing to engineer CIM058-resistant human CD34\n                  <jats:sup>+<\/jats:sup>\n                  HSPCs. When combined, CIM058 and the epitope engineered CD34\n                  <jats:sup>+<\/jats:sup>\n                  HSPCs ameliorated disease phenotype in a \u03b2-thalassemia model. Our results suggest that this approach may overcome the reliance on busulfan or other myeloablative conditioning regimens with their associated morbidities, and by enabling toxin-free conditioning and in vivo selection of edited cells, may facilitate clinical implementation of these highly valuable genetic therapies.\n                <\/jats:p>","DOI":"10.64898\/2026.07.07.736903","type":"posted-content","created":{"date-parts":[[2026,7,8]],"date-time":"2026-07-08T14:05:19Z","timestamp":1783519519000},"source":"Crossref","is-referenced-by-count":0,"title":["CD117 epitope-shielded hematopoietic stem cell transplantation with toxin-free conditioning and in vivo selection ameliorates a \u03b2-thalassemia model"],"prefix":"10.64898","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-1474-1689","authenticated-orcid":false,"given":"Romina","family":"Marone","sequence":"first","affiliation":[{"name":"Department of Biomedicine, Basel University Hospital and University of Basel, Hebelstrasse 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Greece"},{"name":"Aristotle University, Thessaloniki, Greece"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3253-1984","authenticated-orcid":false,"given":"Jessica","family":"Zuin","sequence":"additional","affiliation":[{"name":"Department of Biomedicine, Basel University Hospital and University of Basel, Hebelstrasse 20, CH-4031 Basel, Switzerland"},{"name":"Transplantation Immunology & Nephrology, Basel University Hospital, Petersgraben 4, CH-4031 Basel, Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0009-2484-3495","authenticated-orcid":false,"given":"Alessandro","family":"Sinopoli","sequence":"additional","affiliation":[{"name":"Ridgeline Discovery GmbH, Aeschenvorstadt 36, CH-4051, 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