In utero nanoparticle delivery for site-specific genome editing

Adele S. Ricciardi; Raman Bahal; James S. Farrelly; Elias Quijano; Anthony H. Bianchi; Valerie L. Luks; Rachael Putman; Francesc López-Giráldez; Süleyman Coşkun; Eric Song; Yanfeng Liu; Wei-Che Hsieh; Danith H. Ly; David H. Stitelman; Peter M. Glazer; W. Mark Saltzman

doi:10.1038/s41467-018-04894-2

Nature Communications (Jun 2018)

In utero nanoparticle delivery for site-specific genome editing

Adele S. Ricciardi,
Raman Bahal,
James S. Farrelly,
Elias Quijano,
Anthony H. Bianchi,
Valerie L. Luks,
Rachael Putman,
Francesc López-Giráldez,
Süleyman Coşkun,
Eric Song,
Yanfeng Liu,
Wei-Che Hsieh,
Danith H. Ly,
David H. Stitelman,
Peter M. Glazer,
W. Mark Saltzman

Affiliations

Adele S. Ricciardi: Department of Biomedical Engineering, Yale University
Raman Bahal: Department of Biomedical Engineering, Yale University
James S. Farrelly: Department of Surgery, Yale University
Elias Quijano: Department of Biomedical Engineering, Yale University
Anthony H. Bianchi: Department of Biomedical Engineering, Yale University
Valerie L. Luks: Department of Surgery, Yale University
Rachael Putman: Department of Biomedical Engineering, Yale University
Francesc López-Giráldez: Department of Genetics, Yale University
Süleyman Coşkun: Department of Neurosurgery, Yale University
Eric Song: Department of Biomedical Engineering, Yale University
Yanfeng Liu: Department of Therapeutic Radiology, Yale University
Wei-Che Hsieh: Department of Chemistry and Center for Nucleic Acids Science and Technology (CNAST), Carnegie Mellon University
Danith H. Ly: Department of Chemistry and Center for Nucleic Acids Science and Technology (CNAST), Carnegie Mellon University
David H. Stitelman: Department of Surgery, Yale University
Peter M. Glazer: Department of Therapeutic Radiology, Yale University
W. Mark Saltzman: Department of Biomedical Engineering, Yale University

DOI: https://doi.org/10.1038/s41467-018-04894-2
Journal volume & issue: Vol. 9, no. 1
pp. 1 – 11

Abstract

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The correction of genetic defects in utero could allow for improved outcomes of gene therapy. Here, the authors demonstrate safe delivery of nanoparticles to fetal mouse tissues, and show that nanoparticles containing peptide nucleic acids to edit the beta-globin gene are effective in a mouse model of beta-thalassemia.

Published in Nature Communications

ISSN: 2041-1723 (Online)
Publisher: Nature Portfolio
Country of publisher: United Kingdom
LCC subjects: Science
Website: https://www.nature.com/ncomms/

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