Delivery of CRISPR-Cas tools for in vivo genome editing therapy: Trends and challenges
Open accessAbstract
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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The activity and fidelity of gene editing is heavily affected by the expression level and duration of the editors in the cells.
“The activity and fidelity of gene editing is heavily affected by the expression level and duration of the editors in the cells.”
From Off-target effects in CRISPR/Cas9 gene editing (2023) · cites this paper as Taha et al., 2022
Section 6.4 states that off-target mutagenesis risk depends on the level and duration of Cas9/gRNA expression, supporting the fidelity part of the claim. Section 3.7 discusses expression duration and editing kinetics, but says short-lived editors are usually sufficient for lasting effects; it does not establish that editing activity is heavily affected by both expression level and duration across editors. Page numbers are unavailable in our copy. (LLM, conf=0.83)
Experimental — accuracy not yet measuredMethods to deliver Cas9/sgRNA into target cells profoundly influence its off-target effect.
“Therefore, methods to deliver Cas9/sgRNA into the target cells profoundly influence its off-target effect”
From Off-target effects in CRISPR/Cas9 gene editing (2023) · cites this paper as Taha et al., 2022
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Gene Therapy: Towards a New Era of Medicine
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O’Connor, T. N., van den Bersselaar, L. R., Chen, Y. S., Nicolau, S., Simon, B., Huseth, A., Todd, J. J., Petegem, F. V., Sárközy, A., Goldberg, M. F., et al. (2022) · Journal of Neuromuscular Diseases · doi:10.3233/jnd-221609 · Cited by 14 · see citing works
Current and Emerging Issues in Adeno-Associated Virus Vector-Mediated Liver-Directed Gene Therapy
Piccolo, P., & Brunetti‐Pierri, N. (2024) · Human Gene Therapy · doi:10.1089/hum.2024.179 · Cited by 14 · see citing works
BSA-PEI Nanoparticle Mediated Efficient Delivery of CRISPR/Cas9 into MDA-MB-231 Cells
Rahimi, H., Zaboli, K. A., Thekkiniath, J., Mousavi, S. H., Johari, B., Hashemi, M. R., Nosrati, H., Goldschneider, D., Bernet, A., Danafar, H., et al. (2022) · Molecular Biotechnology · doi:10.1007/s12033-022-00514-z · Cited by 14 · see citing works
Role of CRISPR/Cas9 in the treatment of Duchenne muscular dystrophy and its delivery strategies
Agrawal, P., Harish, V., Mohd, S., Singh, S. K., Tewari, D., Tatiparthi, R., Harshita, Vishwas, S., Sutrapu, S., Dua, K., et al. (2023) · Life Sciences · doi:10.1016/j.lfs.2023.122003 · Cited by 14 · see citing works
Advances in liposome-based delivery of RNA therapeutics for cancer treatment
Sanati, M., Afshari, A. R., Ahmadi, S. S., Kesharwani, P., & Sahebkar, A. (2024) · Progress in molecular biology and translational science · doi:10.1016/bs.pmbts.2023.12.010 · Cited by 14 · see citing works
Non-Viral CRISPR carriers: transient delivery with lasting effects
Lummerstorfer, M., & Lächelt, U. (2026) · Drug Delivery · doi:10.1080/10717544.2026.2614125 · Cited by 13 · see citing works
Genetic therapies and potential therapeutic applications of CRISPR activators in the eye
Ng, B., Kaukonen, M. K., McClements, M. E., Shamsnajafabadi, H., MacLaren, R. E., & Cehajic‐Kapetanovic, J. (2024) · Progress in Retinal and Eye Research · doi:10.1016/j.preteyeres.2024.101289 · Cited by 13 · see citing works
Kaur, G., Arora, J., Sodhi, A. S., Bhatia, S., & Batra, N. (2024) · Molecular Biotechnology · doi:10.1007/s12033-024-01301-8 · Cited by 14 · see citing works
Prime Editing: The Next Frontier in Precision Gene Therapy
Aliciaslan, M., Erbasan, E., Erendor, F., & Şanlıoğlu, S. (2025) · The Journal of Gene Medicine · doi:10.1002/jgm.70040 · Cited by 13 · see citing works
Navigating the CRISPR/Cas Landscape for Enhanced Diagnosis and Treatment of Wilson’s Disease
Choi, W., Cha, S., & Kim, K. (2024) · Cells · doi:10.3390/cells13141214 · Cited by 12 · see citing works
Vectors in CRISPR Gene Editing for Neurological Disorders: Challenges and Opportunities
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Current and emerging therapies for endometriosis-associated pain: a review
Tijani, E. O., Ajayi, L. O., Tijani, O. S., Akano, O. P., & Ajayi, A. F. (2025) · Middle East Fertility Society Journal · doi:10.1186/s43043-025-00221-0 · Cited by 14 · see citing works
Gholamian, S., Baghaee, P., & Doroudian, M. (2025) · Advanced Therapeutics · doi:10.1002/adtp.202400412 · Cited by 11 · see citing works
The history, use, and challenges of therapeutic somatic cell and germline gene editing
Ryu, J., Adashi, E. Y., & Hennebold, J. D. (2023) · Fertility and Sterility · doi:10.1016/j.fertnstert.2023.02.040 · Cited by 12 · see citing works
Liposome‐Enabled Nanomaterials for Muscle Regeneration
Wu, S., & Lu, J. (2025) · Small Methods · doi:10.1002/smtd.202402154 · Cited by 11 · see citing works
- Authors
- Taha, E. A., Lee, J., & Hotta, A.
- Affiliations
- Ain Shams University (EG)
- Kyoto University (JP)
- Corresponding author
- Hotta, Akitsu
- Venue
- Journal of Controlled Release
- Publisher
- Elsevier BV
- Published
- 2022-02
- Volume
- 342
- Pages
- 345-361
- ISSN
- 0168-3659 (ISSN-L)
- 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
- 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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