دورية أكاديمية
Targeted nonviral delivery of genome editors in vivo.
| العنوان: | Targeted nonviral delivery of genome editors in vivo. |
|---|---|
| المؤلفون: | Tsuchida CA; University of California, Berkeley-University of California, San Francisco Graduate Program in Bioengineering, University of California, Berkeley, CA 94720.; Innovative Genomics Institute, University of California, Berkeley, CA 94720., Wasko KM; Innovative Genomics Institute, University of California, Berkeley, CA 94720.; Department of Molecular and Cell Biology, University of California, Berkeley, CA 94720., Hamilton JR; Innovative Genomics Institute, University of California, Berkeley, CA 94720.; Department of Molecular and Cell Biology, University of California, Berkeley, CA 94720., Doudna JA; University of California, Berkeley-University of California, San Francisco Graduate Program in Bioengineering, University of California, Berkeley, CA 94720.; Innovative Genomics Institute, University of California, Berkeley, CA 94720.; Department of Molecular and Cell Biology, University of California, Berkeley, CA 94720.; Department of Chemistry, University of California, Berkeley, CA 94720.; California Institute for Quantitative Biosciences, University of California, Berkeley, CA 94720.; Molecular Biophysics and Integrated Bioimaging Division, Lawrence Berkeley National Laboratory, Berkeley, CA 94720.; Gladstone Institutes, University of California, San Francisco, CA 94158.; HHMI, University of California, Berkeley, CA 94720. |
| المصدر: | Proceedings of the National Academy of Sciences of the United States of America [Proc Natl Acad Sci U S A] 2024 Mar 12; Vol. 121 (11), pp. e2307796121. Date of Electronic Publication: 2024 Mar 04. |
| نوع المنشور: | Review; Journal Article |
| اللغة: | English |
| بيانات الدورية: | Publisher: National Academy of Sciences Country of Publication: United States NLM ID: 7505876 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1091-6490 (Electronic) Linking ISSN: 00278424 NLM ISO Abbreviation: Proc Natl Acad Sci U S A Subsets: MEDLINE |
| أسماء مطبوعة: | Original Publication: Washington, DC : National Academy of Sciences |
| مواضيع طبية MeSH: | Commerce* , Gene Editing*, Genetic Therapy ; RNA, Messenger ; Ribonucleoproteins |
| مستخلص: | Cell-type-specific in vivo delivery of genome editing molecules is the next breakthrough that will drive biological discovery and transform the field of cell and gene therapy. Here, we discuss recent advances in the delivery of CRISPR-Cas genome editors either as preassembled ribonucleoproteins or encoded in mRNA. Both strategies avoid pitfalls of viral vector-mediated delivery and offer advantages including transient editor lifetime and potentially streamlined manufacturing capability that are already proving valuable for clinical use. We review current applications and future opportunities of these emerging delivery approaches that could make genome editing more efficacious and accessible in the future. Competing Interests: Competing interests statement:J.A.D. is a co-founder of Editas Medicine, Intellia Therapeutics, Caribou Biosciences, Mammoth Biosciences, and Scribe Therapeutics. J.R.H. and J.A.D. are co-founders of Azalea Therapeutics. J.A.D is a scientific advisory board member of Intellia Therapeutics, Caribou Biosciences, Mammoth Biosciences, Scribe Therapeutics, Vertex Pharmaceuticals, eFFECTOR Therapeutics, Felix Biosciences, The Column Group, Inari, and Isomorphic Labs. J.A.D. is the Chief Science Advisor at Sixth Street, an advisor at Tempus, and a Director at Johnson & Johnson and Altos Labs. The Regents of the University of California have patents issued and/or pending related to CRISPR technologies and delivery technologies on which C.A.T., J.R.H., and J.A.D. are listed inventors. J.A.D. has sponsored research projects through Biogen, Pfizer, Apple Tree Partners, Genentech, and Roche. |
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| معلومات مُعتمدة: | F31 HL156468 United States HL NHLBI NIH HHS; K99 GM143461 United States GM NIGMS NIH HHS; RM1 HG009490 United States HG NHGRI NIH HHS; U01 AI142817 United States AI NIAID NIH HHS |
| فهرسة مساهمة: | Keywords: CRISPR-Cas; genome editing; in vivo delivery; nonviral delivery; targeted delivery |
| المشرفين على المادة: | 0 (RNA, Messenger) 0 (Ribonucleoproteins) |
| تواريخ الأحداث: | Date Created: 20240304 Date Completed: 20240306 Latest Revision: 20240320 |
| رمز التحديث: | 20240320 |
| مُعرف محوري في PubMed: | PMC10945750 |
| DOI: | 10.1073/pnas.2307796121 |
| PMID: | 38437567 |
| قاعدة البيانات: | MEDLINE |
| تدمد: | 1091-6490 |
|---|---|
| DOI: | 10.1073/pnas.2307796121 |