دورية أكاديمية
أعرض في EDS

Nonviral In Vivo Delivery of CRISPR-Cas9 Using Protein-Agnostic, High-Loading Porous Silicon and Polymer Nanoparticles.

التفاصيل البيبلوغرافية
العنوان: Nonviral In Vivo Delivery of CRISPR-Cas9 Using Protein-Agnostic, High-Loading Porous Silicon and Polymer Nanoparticles.
المؤلفون: Fletcher RB; Department of Biomedical Engineering, Vanderbilt University, Nashville, Tennessee 37235-1631, United States., Stokes LD; Department of Biomedical Engineering, Vanderbilt University, Nashville, Tennessee 37235-1631, United States., Kelly IB 3rd; Department of Biomedical Engineering, Vanderbilt University, Nashville, Tennessee 37235-1631, United States., Henderson KM; Department of Biomedical Engineering, Vanderbilt University, Nashville, Tennessee 37235-1631, United States., Vallecillo-Viejo IC; Department of Biomedical Engineering, Vanderbilt University, Nashville, Tennessee 37235-1631, United States., Colazo JM; Department of Biomedical Engineering, Vanderbilt University, Nashville, Tennessee 37235-1631, United States., Wong BV; Department of Biomedical Engineering, Vanderbilt University, Nashville, Tennessee 37235-1631, United States., Yu F; Department of Biomedical Engineering, Vanderbilt University, Nashville, Tennessee 37235-1631, United States., d'Arcy R; Department of Biomedical Engineering, Vanderbilt University, Nashville, Tennessee 37235-1631, United States., Struthers MN; Department of Biomedical Engineering, Vanderbilt University, Nashville, Tennessee 37235-1631, United States., Evans BC; Department of Biomedical Engineering, Vanderbilt University, Nashville, Tennessee 37235-1631, United States., Ayers J; Department of Biomedical Engineering, Vanderbilt University, Nashville, Tennessee 37235-1631, United States., Castanon M; Department of Biomedical Engineering, Vanderbilt University, Nashville, Tennessee 37235-1631, United States., Weirich MJ; Department of Biomedical Engineering, Vanderbilt University, Nashville, Tennessee 37235-1631, United States., Reilly SK; Department of Biomedical Engineering, Vanderbilt University, Nashville, Tennessee 37235-1631, United States., Patel SS; Department of Biomedical Engineering, Vanderbilt University, Nashville, Tennessee 37235-1631, United States., Ivanova YI; Department of Biomedical Engineering, Vanderbilt University, Nashville, Tennessee 37235-1631, United States., Silvera Batista CA; Department of Chemical and Biomolecular Engineering, Vanderbilt University, Nashville, Tennessee 37235-1631, United States., Weiss SM; Department of Electrical Engineering, Vanderbilt University, Nashville, Tennessee 37235-1631, United States., Gersbach CA; Department of Biomedical Engineering, Duke University, Durham, North Carolina 27708, United States., Brunger JM; Department of Biomedical Engineering, Vanderbilt University, Nashville, Tennessee 37235-1631, United States., Duvall CL; Department of Biomedical Engineering, Vanderbilt University, Nashville, Tennessee 37235-1631, United States.
المصدر: ACS nano [ACS Nano] 2023 Sep 12; Vol. 17 (17), pp. 16412-16431. Date of Electronic Publication: 2023 Aug 15.
نوع المنشور: Journal Article; Research Support, N.I.H., Extramural
اللغة: English
بيانات الدورية: Publisher: American Chemical Society Country of Publication: United States NLM ID: 101313589 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1936-086X (Electronic) Linking ISSN: 19360851 NLM ISO Abbreviation: ACS Nano Subsets: MEDLINE
أسماء مطبوعة: Original Publication: Washington D.C. : American Chemical Society
مواضيع طبية MeSH: CRISPR-Cas Systems*/genetics , Nanoparticles*, Mice ; Animals ; Silicon ; Porosity ; Polymers
مستخلص: The complexity of CRISPR machinery is a challenge to its application for nonviral in vivo therapeutic gene editing. Here, we demonstrate that proteins, regardless of size or charge, efficiently load into porous silicon nanoparticles (PSiNPs). Optimizing the loading strategy yields formulations that are ultrahigh loading─>40% cargo by volume─and highly active. Further tuning of a polymeric coating on the loaded PSiNPs yields nanocomposites that achieve colloidal stability under cryopreservation, endosome escape, and gene editing efficiencies twice that of the commercial standard Lipofectamine CRISPRMAX. In a mouse model of arthritis, PSiNPs edit cells in both the cartilage and synovium of knee joints, and achieve 60% reduction in expression of the therapeutically relevant MMP13 gene. Administered intramuscularly, they are active over a broad dose range, with the highest tested dose yielding nearly 100% muscle fiber editing at the injection site. The nanocomposite PSiNPs are also amenable to systemic delivery. Administered intravenously in a model that mimics muscular dystrophy, they edit sites of inflamed muscle. Collectively, the results demonstrate that the PSiNP nanocomposites are a versatile system that can achieve high loading of diverse cargoes and can be applied for gene editing in both local and systemic delivery applications.
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معلومات مُعتمدة: R01 CA224241 United States CA NCI NIH HHS; R21 AR078636 United States AR NIAMS NIH HHS; R38 HL143619 United States HL NHLBI NIH HHS; U01 AI146356 United States AI NIAID NIH HHS
فهرسة مساهمة: Keywords: CRISPR; Duchenne’s muscular dystrophy; arthritis; porous silicon nanoparticles; ribonucleoprotein
المشرفين على المادة: Z4152N8IUI (Silicon)
0 (Polymers)
تواريخ الأحداث: Date Created: 20230815 Date Completed: 20230914 Latest Revision: 20240529
رمز التحديث: 20240529
مُعرف محوري في PubMed: PMC11129837
DOI: 10.1021/acsnano.2c12261
PMID: 37582231
قاعدة البيانات: MEDLINE
الوصف
تدمد:1936-086X
DOI:10.1021/acsnano.2c12261