In utero delivery of targeted ionizable lipid nanoparticles facilitates in vivo gene editing of hematopoietic stem cells.

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Title: In utero delivery of targeted ionizable lipid nanoparticles facilitates in vivo gene editing of hematopoietic stem cells.
Authors: Palanki R; Department of Bioengineering, University of Pennsylvania, Philadelphia, PA 19104.; Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA 19104., Riley JS; Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA 19104., Bose SK; Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA 19104., Luks V; Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA 19104., Dave A; Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA 19104., Kus N; Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA 19104., White BM; Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA 19104., Ricciardi AS; Department of Bioengineering, University of Pennsylvania, Philadelphia, PA 19104.; Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA 19104., Swingle KL; Department of Bioengineering, University of Pennsylvania, Philadelphia, PA 19104., Xue L; Department of Bioengineering, University of Pennsylvania, Philadelphia, PA 19104., Sung D; Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104., Thatte AS; Department of Bioengineering, University of Pennsylvania, Philadelphia, PA 19104., Safford HC; Department of Bioengineering, University of Pennsylvania, Philadelphia, PA 19104., Chaluvadi VS; Department of Psychiatry, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104., Carpenter M; Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA 19104., Han EL; Department of Bioengineering, University of Pennsylvania, Philadelphia, PA 19104., Maganti R; Department of Bioengineering, University of Pennsylvania, Philadelphia, PA 19104.; Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA 19104., Hamilton AG; Department of Bioengineering, University of Pennsylvania, Philadelphia, PA 19104., Mrksich K; Department of Bioengineering, University of Pennsylvania, Philadelphia, PA 19104., Billingsley MB; Department of Bioengineering, University of Pennsylvania, Philadelphia, PA 19104., Zoltick PW; Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA 19104., Alameh MG; Department of Pathology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104., Weissman D; Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104., Mitchell MJ; Department of Bioengineering, University of Pennsylvania, Philadelphia, PA 19104., Peranteau WH; Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA 19104.
Source: Proceedings of the National Academy of Sciences of the United States of America [Proc Natl Acad Sci U S A] 2024 Aug 06; Vol. 121 (32), pp. e2400783121. Date of Electronic Publication: 2024 Jul 30.
Publication Type: Journal Article
Journal Info: Publisher: National Academy of Sciences Country of Publication: United States NLM ID: 7505876 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1091-6490 (Electronic) Linking ISSN: 00278424 NLM ISO Abbreviation: Proc Natl Acad Sci U S A Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:1091-6490
DOI:10.1073/pnas.2400783121