Evaluation of subretinally delivered Cas9 ribonucleoproteins in murine and porcine animal models highlights key considerations for therapeutic translation of genetic medicines

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Main Authors: Spencer C. Wei, Aaron J. Cantor, Jack Walleshauser, Rina Mepani, Kory Melton, Ashil Bans, Prachi Khekare, Suhani Gupta, Jonathan Wang, Craig Soares, Radwan Kiwan, Jieun Lee, Shannon McCawley, Vihasi Jani, Weng In Leong, Pawan K. Shahi, Jean Chan, Pierre Boivin, Peter Otoupal, Bikash R. Pattnaik, David M. Gamm, Krishanu Saha, Benjamin G. Gowen, Mary Haak-Frendscho, Mary J. Janatpour, Adam P. Silverman
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2025-01-01
Series:PLoS ONE
Online Access:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12186880/?tool=EBI
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author Spencer C. Wei
Aaron J. Cantor
Jack Walleshauser
Rina Mepani
Kory Melton
Ashil Bans
Prachi Khekare
Suhani Gupta
Jonathan Wang
Craig Soares
Radwan Kiwan
Jieun Lee
Shannon McCawley
Vihasi Jani
Weng In Leong
Pawan K. Shahi
Jean Chan
Pierre Boivin
Peter Otoupal
Bikash R. Pattnaik
David M. Gamm
Krishanu Saha
Benjamin G. Gowen
Mary Haak-Frendscho
Mary J. Janatpour
Adam P. Silverman
author_facet Spencer C. Wei
Aaron J. Cantor
Jack Walleshauser
Rina Mepani
Kory Melton
Ashil Bans
Prachi Khekare
Suhani Gupta
Jonathan Wang
Craig Soares
Radwan Kiwan
Jieun Lee
Shannon McCawley
Vihasi Jani
Weng In Leong
Pawan K. Shahi
Jean Chan
Pierre Boivin
Peter Otoupal
Bikash R. Pattnaik
David M. Gamm
Krishanu Saha
Benjamin G. Gowen
Mary Haak-Frendscho
Mary J. Janatpour
Adam P. Silverman
author_sort Spencer C. Wei
collection DOAJ
format Article
id doaj-art-4c5976beaa5249cdbef17b4a592e1602
institution OA Journals
issn 1932-6203
language English
publishDate 2025-01-01
publisher Public Library of Science (PLoS)
record_format Article
series PLoS ONE
spelling doaj-art-4c5976beaa5249cdbef17b4a592e16022025-08-20T02:22:05ZengPublic Library of Science (PLoS)PLoS ONE1932-62032025-01-01206Evaluation of subretinally delivered Cas9 ribonucleoproteins in murine and porcine animal models highlights key considerations for therapeutic translation of genetic medicinesSpencer C. WeiAaron J. CantorJack WalleshauserRina MepaniKory MeltonAshil BansPrachi KhekareSuhani GuptaJonathan WangCraig SoaresRadwan KiwanJieun LeeShannon McCawleyVihasi JaniWeng In LeongPawan K. ShahiJean ChanPierre BoivinPeter OtoupalBikash R. PattnaikDavid M. GammKrishanu SahaBenjamin G. GowenMary Haak-FrendschoMary J. JanatpourAdam P. Silvermanhttps://www.ncbi.nlm.nih.gov/pmc/articles/PMC12186880/?tool=EBI
spellingShingle Spencer C. Wei
Aaron J. Cantor
Jack Walleshauser
Rina Mepani
Kory Melton
Ashil Bans
Prachi Khekare
Suhani Gupta
Jonathan Wang
Craig Soares
Radwan Kiwan
Jieun Lee
Shannon McCawley
Vihasi Jani
Weng In Leong
Pawan K. Shahi
Jean Chan
Pierre Boivin
Peter Otoupal
Bikash R. Pattnaik
David M. Gamm
Krishanu Saha
Benjamin G. Gowen
Mary Haak-Frendscho
Mary J. Janatpour
Adam P. Silverman
Evaluation of subretinally delivered Cas9 ribonucleoproteins in murine and porcine animal models highlights key considerations for therapeutic translation of genetic medicines
PLoS ONE
title Evaluation of subretinally delivered Cas9 ribonucleoproteins in murine and porcine animal models highlights key considerations for therapeutic translation of genetic medicines
title_full Evaluation of subretinally delivered Cas9 ribonucleoproteins in murine and porcine animal models highlights key considerations for therapeutic translation of genetic medicines
title_fullStr Evaluation of subretinally delivered Cas9 ribonucleoproteins in murine and porcine animal models highlights key considerations for therapeutic translation of genetic medicines
title_full_unstemmed Evaluation of subretinally delivered Cas9 ribonucleoproteins in murine and porcine animal models highlights key considerations for therapeutic translation of genetic medicines
title_short Evaluation of subretinally delivered Cas9 ribonucleoproteins in murine and porcine animal models highlights key considerations for therapeutic translation of genetic medicines
title_sort evaluation of subretinally delivered cas9 ribonucleoproteins in murine and porcine animal models highlights key considerations for therapeutic translation of genetic medicines
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12186880/?tool=EBI
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