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Selection of human hematopoietic stem cells bearing the intended functional edit by transient AND-gate reporters.

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Title: Selection of human hematopoietic stem cells bearing the intended functional edit by transient AND-gate reporters.
Authors: Canarutto D; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Vita-Salute San Raffaele University, Milan, Italy., Fiumara M; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Vita-Salute San Raffaele University, Milan, Italy., Venkatesan V; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Gaddoni C; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Shiroshita K; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Varesi A; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Princess Margaret Cancer Centre, Toronto, Ontario, Canada.; Department of Molecular Genetics, University of Toronto, Toronto, Ontario, Canada., Albano L; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Pelosi G; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Silva A; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Firrito C; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Fondazione Telethon ETS, Rome, Italy., Schiroli G; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Tessera Therapeutics, Sommerville, MA, USA., Cipria D; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Beretta S; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Amabile A; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Icahn Genomics Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA.; RNA Nanoparticle Core, Icahn School of Medicine at Mount Sinai, New York, NY, USA.; Department of Immunology and Immunotherapy, Icahn School of Medicine at Mount Sinai, New York, NY, USA., Villa A; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy., Zonari E; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Gentner B; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Ludwig Institute for Cancer Research and Department of Oncology, University of Lausanne (UNIL), Lausanne, Switzerland.; Lausanne University Hospital (CHUV), Lausanne, Switzerland., Lombardo A; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Vita-Salute San Raffaele University, Milan, Italy., Genovese P; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Division of Hematology/Oncology, Boston Children's Hospital, Boston, MA, USA.; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA, USA.; Harvard Medical School, Boston, MA, USA., Ferrari S; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Vita-Salute San Raffaele University, Milan, Italy., Naldini L; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy. naldini.luigi@hsr.it.; Vita-Salute San Raffaele University, Milan, Italy. naldini.luigi@hsr.it.
Source: Nature biotechnology [Nat Biotechnol] 2026 Jun 01. Date of Electronic Publication: 2026 Jun 01.
Publication Type: Journal Article
Journal Info: Publisher: Nature America Publishing Country of Publication: United States NLM ID: 9604648 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1546-1696 (Electronic) Linking ISSN: 10870156 NLM ISO Abbreviation: Nat Biotechnol Subsets: MEDLINE
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  Data: Selection of human hematopoietic stem cells bearing the intended functional edit by transient AND-gate reporters.
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  Data: <searchLink fieldCode="AU" term="%22Canarutto+D%22">Canarutto D</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Vita-Salute San Raffaele University, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Fiumara+M%22">Fiumara M</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Vita-Salute San Raffaele University, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Venkatesan+V%22">Venkatesan V</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Gaddoni+C%22">Gaddoni C</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Shiroshita+K%22">Shiroshita K</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Varesi+A%22">Varesi A</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Princess Margaret Cancer Centre, Toronto, Ontario, Canada.; Department of Molecular Genetics, University of Toronto, Toronto, Ontario, Canada.<br /><searchLink fieldCode="AU" term="%22Albano+L%22">Albano L</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Pelosi+G%22">Pelosi G</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Silva+A%22">Silva A</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Firrito+C%22">Firrito C</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Fondazione Telethon ETS, Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Schiroli+G%22">Schiroli G</searchLink>; 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San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Zonari+E%22">Zonari E</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Gentner+B%22">Gentner B</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Ludwig Institute for Cancer Research and Department of Oncology, University of Lausanne (UNIL), Lausanne, Switzerland.; Lausanne University Hospital (CHUV), Lausanne, Switzerland.<br /><searchLink fieldCode="AU" term="%22Lombardo+A%22">Lombardo A</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Vita-Salute San Raffaele University, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Genovese+P%22">Genovese P</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Division of Hematology/Oncology, Boston Children's Hospital, Boston, MA, USA.; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA, USA.; Harvard Medical School, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Ferrari+S%22">Ferrari S</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Vita-Salute San Raffaele University, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Naldini+L%22">Naldini L</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy. naldini.luigi@hsr.it.; Vita-Salute San Raffaele University, Milan, Italy. naldini.luigi@hsr.it.
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