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Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing.
Li, Bowen; Manan, Rajith Singh; Liang, Shun-Qing; Gordon, Akiva; Jiang, Allen; Varley, Andrew; Gao, Guangping; Langer, Robert; Xue, Wen; Anderson, Daniel.
Afiliación
  • Li B; David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, USA.
  • Manan RS; Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA, USA.
  • Liang SQ; Department of Pharmaceutical Sciences, Leslie Dan Faculty of Pharmacy, University of Toronto, Toronto, Ontario, Canada.
  • Gordon A; Institute of Biomedical Engineering, University of Toronto, Toronto, Ontario, Canada.
  • Jiang A; David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, USA.
  • Varley A; Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA, USA.
  • Gao G; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA.
  • Langer R; David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, USA.
  • Xue W; Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA, USA.
  • Anderson D; David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, USA.
Nat Biotechnol ; 41(10): 1410-1415, 2023 Oct.
Article en En | MEDLINE | ID: mdl-36997680
ABSTRACT
The expanding applications of nonviral genomic medicines in the lung remain restricted by delivery challenges. Here, leveraging a high-throughput platform, we synthesize and screen a combinatorial library of biodegradable ionizable lipids to build inhalable delivery vehicles for messenger RNA and CRISPR-Cas9 gene editors. Lead lipid nanoparticles are amenable for repeated intratracheal dosing and could achieve efficient gene editing in lung epithelium, providing avenues for gene therapy of congenital lung diseases.
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Texto completo: 1 Banco de datos: MEDLINE Idioma: En Revista: Nat Biotechnol Asunto de la revista: BIOTECNOLOGIA Año: 2023 Tipo del documento: Article País de afiliación: Estados Unidos
Texto completo
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XML
PubMed Links
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Texto completo: 1 Banco de datos: MEDLINE Idioma: En Revista: Nat Biotechnol Asunto de la revista: BIOTECNOLOGIA Año: 2023 Tipo del documento: Article País de afiliación: Estados Unidos