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dc.contributor.authorWei, Tuo
dc.contributor.authorCheng, Qiang
dc.contributor.authorFarbiak, Lukas
dc.contributor.authorAnderson, Daniel G
dc.contributor.authorLanger, Robert
dc.contributor.authorSiegwart, Daniel J
dc.date.accessioned2022-01-20T15:16:04Z
dc.date.available2021-10-27T19:53:11Z
dc.date.available2022-01-20T15:16:04Z
dc.date.issued2020-07
dc.identifier.issn1936-086X
dc.identifier.issn1936-0851
dc.identifier.urihttps://hdl.handle.net/1721.1/133499.2
dc.description.abstractCopyright © 2020 American Chemical Society. CRISPR/Cas9-based genome editing has quickly emerged as a powerful breakthrough technology for use in diverse settings across biomedical research and therapeutic development. Recent efforts toward understanding gene modification methods in vitro have led to substantial improvements in ex vivo genome editing efficiency. Because disease targets for genomic correction are often localized in specific organs, realization of the full potential of genomic medicines will require delivery of CRISPR/Cas9 systems targeting specific tissues and cells directly in vivo. In this Perspective, we focus on progress toward in vivo delivery of CRISPR/Cas components. Viral and nonviral delivery systems are both promising for gene editing in diverse tissues via local injection and systemic injection. We describe the various viral vectors and synthetic nonviral materials used for in vivo gene editing and applications to research and therapeutic models, and summarize opportunities and progress to date for both methods. We also discuss challenges for viral delivery, including overcoming limited packaging capacity, immunogenicity associated with multiple dosing, and the potential for off-target effects, and nonviral delivery, including efforts to increase efficacy and to expand utility of nonviral carriers for use in extrahepatic tissues and cancer. Looking ahead, additional advances in the safety and efficiency of viral and nonviral delivery systems for tissue- and cell-type-specific gene editing will be required to enable broad clinical translation. We provide a summary of current delivery systems used for in vivo genome editing, organized with respect to route of administration, and highlight immediate opportunities for biomedical research and applications. Furthermore, we discuss current challenges for in vivo delivery of CRISPR/Cas9 systems to guide the development of future therapies.en_US
dc.language.isoen
dc.publisherAmerican Chemical Society (ACS)en_US
dc.relation.isversionofhttp://dx.doi.org/10.1021/ACSNANO.0C04707en_US
dc.rightsCreative Commons Attribution-Noncommercial-Share Alikeen_US
dc.rights.urihttps://creativecommons.org/licenses/by/4.0/en_US
dc.sourcePMCen_US
dc.titleDelivery of Tissue-Targeted Scalpels: Opportunities and Challenges for In Vivo CRISPR/Cas-Based Genome Editingen_US
dc.typeArticleen_US
dc.contributor.departmentMassachusetts Institute of Technology. Department of Chemical Engineering
dc.contributor.departmentKoch Institute for Integrative Cancer Research at MIT
dc.contributor.departmentHarvard University--MIT Division of Health Sciences and Technology
dc.contributor.departmentMassachusetts Institute of Technology. Institute for Medical Engineering & Science
dc.relation.journalACS Nanoen_US
dc.eprint.versionAuthor's final manuscripten_US
dc.type.urihttp://purl.org/eprint/type/JournalArticleen_US
eprint.statushttp://purl.org/eprint/status/PeerRevieweden_US
dc.date.updated2021-08-27T12:51:09Z
dspace.orderedauthorsWei, T; Cheng, Q; Farbiak, L; Anderson, DG; Langer, R; Siegwart, DJen_US
dspace.date.submission2021-08-27T12:51:11Z
mit.journal.volume14en_US
mit.journal.issue8en_US
mit.licenseOPEN_ACCESS_POLICY
mit.metadata.statusAuthority Work Neededen_US


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