Therapeutic in vivo delivery of gene editing agents
In vivo gene editing therapies offer the potential to treat the root causes of many genetic diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to safely and efficiently deliver gene editing agents to relevant organs and tissues in vivo. Here, we review current d...
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| Veröffentlicht in: | Cell 2022-07, Vol.185 (15), p.2806-2827 |
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| container_title | Cell |
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| creator | Raguram, Aditya Banskota, Samagya Liu, David R. |
| description | In vivo gene editing therapies offer the potential to treat the root causes of many genetic diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to safely and efficiently deliver gene editing agents to relevant organs and tissues in vivo. Here, we review current delivery technologies that have been used to enable therapeutic in vivo gene editing, including viral vectors, lipid nanoparticles, and virus-like particles. Since no single delivery modality is likely to be appropriate for every possible application, we compare the benefits and drawbacks of each method and highlight opportunities for future improvements.
Therapeutic gene editing has made exciting progress in the past few years, in part through key advances in in vivo delivery technologies. David Liu and colleagues review the essential characteristics required for efficient in vivo gene editing delivery vehicles, discussing the pros and cons of viral vectors, lipid nanoparticles, and virus-like particles. |
| doi_str_mv | 10.1016/j.cell.2022.03.045 |
| format | Article |
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Therapeutic gene editing has made exciting progress in the past few years, in part through key advances in in vivo delivery technologies. David Liu and colleagues review the essential characteristics required for efficient in vivo gene editing delivery vehicles, discussing the pros and cons of viral vectors, lipid nanoparticles, and virus-like particles.</description><subject>CRISPR-Cas Systems - genetics</subject><subject>Gene Editing - methods</subject><subject>genes</subject><subject>Genetic Therapy - methods</subject><subject>Genetic Vectors</subject><subject>lipids</subject><subject>Liposomes</subject><subject>Nanoparticles</subject><subject>therapeutics</subject><issn>0092-8674</issn><issn>1097-4172</issn><issn>1097-4172</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2022</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNqFkc1O3DAUha0KVAboC3RRZckm4dqOY0dCSAi1FAmJDawt_9wMHmWSwc5EmrfhWfpkJBqKYNOurCuf8137HEK-Uygo0Op8VThs24IBYwXwAkrxhSwo1DIvqWQHZAFQs1xVsjwixymtAEAJIb6SIy5krQCqBSkfnjCaDW6H4LLQ_XkZw9hnHtswYtxlfZMtscMMfRhCt8zMNA3plBw2pk347e08IY-_fj5c_87v7m9ur6_uclcqNeTeAZeuro2AipXUoRKeOkYtBSa8tZYb2TSOqgZqL8BSxtBRaw04a7ww_IRc7rmbrV2jd9PuaFq9iWFt4k73JujPN1140st-1HUpSs7lBDh7A8T-eYtp0OuQ5tBMh_02aSapYpXiFfxfWikpGZVyprK91MU-pYjN-4so6LkZvdKzU8_NaOB6amYy_fj4l3fL3yomwcVegFOiY8CokwvYuSn6iG7Qvg__4r8Cu9ChCw</recordid><startdate>20220721</startdate><enddate>20220721</enddate><creator>Raguram, Aditya</creator><creator>Banskota, Samagya</creator><creator>Liu, David R.</creator><general>Elsevier Inc</general><scope>6I.</scope><scope>AAFTH</scope><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7X8</scope><scope>7S9</scope><scope>L.6</scope><scope>5PM</scope><orcidid>https://orcid.org/0000-0002-9943-7557</orcidid></search><sort><creationdate>20220721</creationdate><title>Therapeutic in vivo delivery of gene editing agents</title><author>Raguram, Aditya ; Banskota, Samagya ; Liu, David R.</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c488t-dc037c99a506241ce85d1c21b1025dbbb3a7ffc18f09d50b122ec1bba0cbad5a3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2022</creationdate><topic>CRISPR-Cas Systems - genetics</topic><topic>Gene Editing - methods</topic><topic>genes</topic><topic>Genetic Therapy - methods</topic><topic>Genetic Vectors</topic><topic>lipids</topic><topic>Liposomes</topic><topic>Nanoparticles</topic><topic>therapeutics</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Raguram, Aditya</creatorcontrib><creatorcontrib>Banskota, Samagya</creatorcontrib><creatorcontrib>Liu, David R.</creatorcontrib><collection>ScienceDirect Open Access Titles</collection><collection>Elsevier:ScienceDirect:Open Access</collection><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>MEDLINE - Academic</collection><collection>AGRICOLA</collection><collection>AGRICOLA - Academic</collection><collection>PubMed Central (Full Participant titles)</collection><jtitle>Cell</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Raguram, Aditya</au><au>Banskota, Samagya</au><au>Liu, David R.</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Therapeutic in vivo delivery of gene editing agents</atitle><jtitle>Cell</jtitle><addtitle>Cell</addtitle><date>2022-07-21</date><risdate>2022</risdate><volume>185</volume><issue>15</issue><spage>2806</spage><epage>2827</epage><pages>2806-2827</pages><issn>0092-8674</issn><issn>1097-4172</issn><eissn>1097-4172</eissn><abstract>In vivo gene editing therapies offer the potential to treat the root causes of many genetic diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to safely and efficiently deliver gene editing agents to relevant organs and tissues in vivo. Here, we review current delivery technologies that have been used to enable therapeutic in vivo gene editing, including viral vectors, lipid nanoparticles, and virus-like particles. Since no single delivery modality is likely to be appropriate for every possible application, we compare the benefits and drawbacks of each method and highlight opportunities for future improvements.
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| language | eng |
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| source | MEDLINE; Cell Press Free Archives; Elsevier ScienceDirect Journals; Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals |
| subjects | CRISPR-Cas Systems - genetics Gene Editing - methods genes Genetic Therapy - methods Genetic Vectors lipids Liposomes Nanoparticles therapeutics |
| title | Therapeutic in vivo delivery of gene editing agents |
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