Cited by 259CRISPR and Genetic EngineeringRNA Interference and Gene DeliveryVirus-based gene therapy research

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Delivery of CRISPR-Cas tools for in vivo genome editing therapy: Trends and challenges

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Taha, Eman A., Lee, Joseph, Hotta, Akitsu·2022·Journal of Controlled Release

DOI 10.1016/j.jconrel.2022.01.013

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Abstract

The discovery of clustered regularly interspaced short palindromic repeats (CRISPR) genome editing technology opened the door to provide a versatile approach for treating multiple diseases. Promising results have been shown in numerous pre-clinical studies and clinical trials. However, a safe and effective method to deliver genome-editing components is still a key challenge for in vivo genome editing therapy. Adeno-associated virus (AAV) is one of the most commonly used vector systems to date, but immunogenicity against capsid, liver toxicity at high dose, and potential genotoxicity caused by off-target mutagenesis and genomic integration remain unsolved. Recently developed transient delivery systems, such as virus-like particle (VLP) and lipid nanoparticle (LNP), may solve some of the issues. This review summarizes existing in vivo delivery systems and possible solutions to overcome their limitations. Also, we highlight the ongoing clinical trials for in vivo genome editing therapy and recently developed genome editing tools for their potential applications.

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3 of the 180 references couldn’t be matched to a known paper.

Research works printed but not linked: 5

Audentes press release
Audentes press release
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See all 259

Authors: Taha, E. A., Lee, J., & Hotta, A.
Affiliations: Ain Shams University (EG)
Kyoto University (JP)
Corresponding author: Hotta, Akitsu
ORCID: Taha, Eman A.
Lee, Joseph
Hotta, Akitsu
Venue: Journal of Controlled Release
Publisher: Elsevier BV
Published: 2022-02
Volume: 342
Pages: 345-361
ISSN: 0168-3659 (ISSN-L)
DOI: 10.1016/j.jconrel.2022.01.013
OpenAlex: W4205237319
Cited by: 259
References: 180
Citations by year: 2026: 48 · 2025: 72 · 2024: 70 · 2023: 51 · 2022: 18
FWCI: 5.49
Type: journal article
Language: en
Open access: hybrid
doi.org
Licence: cc-by
www.elsevier.com/tdm/userlicense/1.0/ (tdm)
www.elsevier.com/legal/tdmrep-license (tdm)
creativecommons.org/licenses/by/4.0/ (vor)
Locations: Journal of Controlled Release (publishedVersion, cc-by)
PubMed (publishedVersion)
Institutional Repositories DataBase (IRDB) (submittedVersion, cc-by)
Kyoto University Research Information Repository (Kyoto University) (submittedVersion, cc-by)
Greater South Information System
Funders: Takeda Pharmaceutical Co Ltd
Japan Agency for Medical Research and Development (JP21bm0804005, JP21im0210115, JP19ek0109293, JP21bm0104001)
Japan Science and Technology Agency (19H02924)
National Center of Neurology and Psychiatry (28-6)
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
Keywords: CRISPR-Cas, in vivo gene editing, gene delivery, adeno-associated virus vectors, virus-like particles, lipid nanoparticles, immunogenicity, hepatotoxicity, off-target mutagenesis, chromosomal integration, clinical trials, genome editing tools
MeSH terms: Gene Editing, Liposomes, Nanoparticles, CRISPR-Cas Systems

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Delivery of CRISPR-Cas tools for in vivo genome editing therapy: Trends and challenges

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