Delivery strategies for CRISPR/Cas genome editing tool for retinal dystrophies: Challenges and opportunities
Open accessAbstract
CRISPR/Cas, an adaptive immune system in bacteria, has been adopted as an efficient and precise tool for site-specific gene editing with potential therapeutic opportunities. It has been explored for a variety of applications, including gene modulation, epigenome editing, diagnosis, mRNA editing, etc. It has found applications in retinal dystrophic conditions including progressive cone and cone-rod dystrophies, congenital stationary night blindness, X-linked juvenile retinoschisis, retinitis pigmentosa, age-related macular degeneration, leber's congenital amaurosis, etc. Most of the therapies for retinal dystrophic conditions work by regressing symptoms instead of reversing the gene mutations. CRISPR/Cas9 through indel could impart beneficial effects in the reversal of gene mutations in dystrophic conditions. Recent research has also consolidated on the approaches of using CRISPR systems for retinal dystrophies but their delivery to the posterior part of the eye is a major concern due to high molecular weight, negative charge, and in vivo stability of CRISPR components. Recently, non-viral vectors have gained interest due to their potential in tissue-specific nucleic acid (miRNA/siRNA/CRISPR) delivery. This review highlights the opportunities of retinal dystrophies management using CRISPR/Cas nanomedicine.
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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 Lohia et al., 2022
Methods 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 Lohia et al., 2022
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Wang, H., Wei, F., Li, H., Ji, X., Li, S., & Chen, X. (2012) · International Journal of Molecular Medicine · doi:10.3892/ijmm.2012.1197 · Cited by 16 · see citing works
Genome Surgery and Gene Therapy in Retinal Disorders.
Chan, L., Mahajan, V. B., & Tsang, S. H. (2017) · PubMed Central · Cited by 14 · see citing works
Targeted Genome Editing Using DNA-Free RNA-Guided Cas9 Ribonucleoprotein for CHO Cell Engineering
Shin, J., Lee, N., Cho, S., & Cho, B. (2018) · Methods in molecular biology · doi:10.1007/978-1-4939-7795-6_8 · Cited by 12 · see citing works
Celea, C., Pop, M., Avidis-Zamfiroiu, N., et al. (2015) · PubMed · Cited by 7 · see citing works
Emerging Drug Therapies for Inherited Retinal Dystrophies
Sundaramurthi, H., Moran, A. L., Perpetuini, A. C., Reynolds, A., & Kennedy, B. N. (2019) · Advances in experimental medicine and biology · doi:10.1007/978-3-030-27378-1_43 · Cited by 7 · see citing works
Flagged first, then by citations
Research works printed but not linked: 3
- [33]Colella P. Auricchio A. Gene therapy of inherited retinopathies: a long and successful road from viral vectors to patients Human gene therapy 23 2012 796 807 22734691 PMC3413895
- [88]Fuster-Garcia C. Garcia-Garcia G. Gonzalez-Romero E. Jaijo T. Sequedo M.D. Ayuso C. USH2A gene editing using the CRISPR system Mol Ther Nucl acids 8 2017 529 541 PMC5573797 28918053
- [126]Fu Y. Foden J.A. Khayter C. Maeder M.L. Reyon D. Joung J.K. Sander J.D. Hgh-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells Nat Biotechnol 31 2013 822 826 23792628 PMC3773023
Cited by
Sorted by citations
Off-target effects in CRISPR/Cas9 gene editing
Guo, C., Ma, X., Gao, F., & Guo, Y. (2023) · Frontiers in Bioengineering and Biotechnology · doi:10.3389/fbioe.2023.1143157 · Cited by 625 · see citing works
“Therefore, methods to deliver Cas9/sgRNA into the target cells profoundly influence its off-target effect (Lohia et al., 2022; Taha et al., 2022).”
Method
- Authors
- Lohia, A., Sahel, D. K., Salman, M., Singh, V., Mariappan, I., Mittal, A., & Chitkara, D.
- Affiliations
- Birla Institute of Technology and Science, Pilani (IN)
- L V Prasad Eye Institute (IN)
- Corresponding author
- Chitkara, Deepak
- Venue
- Asian Journal of Pharmaceutical Sciences
- Publisher
- Elsevier BV
- Published
- 2022-03
- Volume
- 17
- Issue
- 2
- Pages
- 153-176
- ISSN
- 1818-0876 (ISSN-L)
- PMC
- PMC9614410
- OpenAlex
- W4212945596
- Cited by
- 33
- References
- 182
- Citations by year
- 2026: 2 · 2025: 9 · 2024: 7 · 2023: 10 · 2022: 5
- FWCI
- 0.81
- Type
- journal article
- Language
- en
- Open access
- diamond
- doi.org
- Licence
- Locations
- Asian Journal of Pharmaceutical Sciences (publishedVersion, cc-by-nc-nd)
- PubMed Central (publishedVersion, cc-by-nc-nd)
- PubMed (publishedVersion)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
- Funders
- Government of India Department of Biotechnology (BT/PR26897/NNT/28/1489/2017)
- Indian Council of Medical Research (3/1/3/JRF-2019/HRD)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Retinal Development and Disorders
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Keywords
- CRISPR/Cas9, retinal dystrophy, gene delivery, non-viral vectors, retinitis pigmentosa, cone-rod dystrophy, congenital stationary night blindness, X-linked retinoschisis, age-related macular degeneration, Leber congenital amaurosis, genome editing
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
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