{"id":"https://openalex.org/W4212945596","doi":"https://doi.org/10.1016/j.ajps.2022.02.001","title":"Delivery strategies for CRISPR/Cas genome editing tool for retinal dystrophies: challenges and opportunities","display_name":"Delivery strategies for CRISPR/Cas genome editing tool for retinal dystrophies: challenges and opportunities","publication_year":2022,"publication_date":"2022-02-13","ids":{"openalex":"https://openalex.org/W4212945596","doi":"https://doi.org/10.1016/j.ajps.2022.02.001","pmid":"https://pubmed.ncbi.nlm.nih.gov/36320315","pmcid":"https://www.ncbi.nlm.nih.gov/pmc/articles/9614410"},"language":"en","primary_location":{"id":"doi:10.1016/j.ajps.2022.02.001","doi":"https://doi.org/10.1016/j.ajps.2022.02.001","is_oa":true,"landing_page_url":"https://doi.org/10.1016/j.ajps.2022.02.001","pdf_url":"https://www.sciencedirect.com/science/article/pii/S1818087622000149/pdf","source":{"id":"https://openalex.org/S2764650913","display_name":"Asian Journal of Pharmaceutical Sciences","issn_l":"1818-0876","issn":["1818-0876","2221-285X"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["cwts-core","doaj","dongbi-c","doyens","jufo-1","ki-jl-1","oarl","poland-100","russia-white-list-1"],"host_organization":"https://openalex.org/P4310320990","host_organization_name":"Elsevier BV","host_organization_lineage":["https://openalex.org/P4310320990"],"host_organization_lineage_names":["Elsevier BV"],"type":"journal"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Asian Journal of Pharmaceutical Sciences","raw_type":"journal-article"},"volume":null,"type":"review","is_traditional":true,"indexed_in":["crossref","doaj","pubmed"],"open_access":{"is_oa":true,"oa_status":"diamond","oa_url":"https://www.sciencedirect.com/science/article/pii/S1818087622000149/pdf","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5075815251","display_name":"Aayushi Lohia","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I74796645","display_name":"Birla Institute of Technology and Science, Pilani","ror":"https://ror.org/001p3jz28","country_code":"IN","type":"education","lineage":["https://openalex.org/I74796645"]}],"countries":["IN"],"is_corresponding":false,"raw_author_name":"Aayushi Lohia","raw_affiliation_strings":["Department of Pharmacy, Birla Institute of Technology and Science, Pilani (BITS-Pilani), Vidya Vihar Campus, Pilani 333031, India"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Pharmacy, Birla Institute of Technology and Science, Pilani (BITS-Pilani), Vidya Vihar Campus, Pilani 333031, India","institution_ids":["https://openalex.org/I74796645"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5091869018","display_name":"Deepak Kumar Sahel","orcid":"https://orcid.org/0009-0003-1199-1540","observed_orcids":["https://orcid.org/0009-0003-1199-1540"]},"institutions":[{"id":"https://openalex.org/I74796645","display_name":"Birla Institute of Technology and Science, Pilani","ror":"https://ror.org/001p3jz28","country_code":"IN","type":"education","lineage":["https://openalex.org/I74796645"]}],"countries":["IN"],"is_corresponding":false,"raw_author_name":"Deepak Kumar Sahel","raw_affiliation_strings":["Department of Pharmacy, Birla Institute of Technology and Science, Pilani (BITS-Pilani), Vidya Vihar Campus, Pilani 333031, India"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Pharmacy, Birla Institute of Technology and Science, Pilani (BITS-Pilani), Vidya Vihar Campus, Pilani 333031, India","institution_ids":["https://openalex.org/I74796645"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5059035346","display_name":"Mohd Salman","orcid":"https://orcid.org/0009-0001-1536-3803","observed_orcids":["https://orcid.org/0009-0001-1536-3803"]},"institutions":[{"id":"https://openalex.org/I2801255070","display_name":"L V Prasad Eye Institute","ror":"https://ror.org/01w8z9742","country_code":"IN","type":"funder","lineage":["https://openalex.org/I2801255070"]}],"countries":["IN"],"is_corresponding":false,"raw_author_name":"Mohd Salman","raw_affiliation_strings":["Prof. Brien Holden Eye Research Center, Champalimaud Translational Centre for Eye Research, L.V. Prasad Eye Institute, Kallam Anji Reddy Campus, Hyderabad 500034, India"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Prof. Brien Holden Eye Research Center, Champalimaud Translational Centre for Eye Research, L.V. Prasad Eye Institute, Kallam Anji Reddy Campus, Hyderabad 500034, India","institution_ids":["https://openalex.org/I2801255070"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5029671824","display_name":"Vivek Singh","orcid":"https://orcid.org/0000-0003-3958-5635","observed_orcids":["https://orcid.org/0000-0003-3958-5635"]},"institutions":[{"id":"https://openalex.org/I2801255070","display_name":"L V Prasad Eye Institute","ror":"https://ror.org/01w8z9742","country_code":"IN","type":"funder","lineage":["https://openalex.org/I2801255070"]}],"countries":["IN"],"is_corresponding":false,"raw_author_name":"Vivek Singh","raw_affiliation_strings":["Prof. Brien Holden Eye Research Center, Champalimaud Translational Centre for Eye Research, L.V. Prasad Eye Institute, Kallam Anji Reddy Campus, Hyderabad 500034, India"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Prof. Brien Holden Eye Research Center, Champalimaud Translational Centre for Eye Research, L.V. Prasad Eye Institute, Kallam Anji Reddy Campus, Hyderabad 500034, India","institution_ids":["https://openalex.org/I2801255070"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5032471166","display_name":"Indumathi Mariappan","orcid":"https://orcid.org/0000-0001-7059-3030","observed_orcids":["https://orcid.org/0000-0001-7059-3030"]},"institutions":[{"id":"https://openalex.org/I2801255070","display_name":"L V Prasad Eye Institute","ror":"https://ror.org/01w8z9742","country_code":"IN","type":"funder","lineage":["https://openalex.org/I2801255070"]}],"countries":["IN"],"is_corresponding":false,"raw_author_name":"Indumathi Mariappan","raw_affiliation_strings":["Prof. Brien Holden Eye Research Center, Champalimaud Translational Centre for Eye Research, L.V. Prasad Eye Institute, Kallam Anji Reddy Campus, Hyderabad 500034, India"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Prof. Brien Holden Eye Research Center, Champalimaud Translational Centre for Eye Research, L.V. Prasad Eye Institute, Kallam Anji Reddy Campus, Hyderabad 500034, India","institution_ids":["https://openalex.org/I2801255070"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5040955433","display_name":"Anupama Mittal","orcid":"https://orcid.org/0000-0003-3344-9579","observed_orcids":["https://orcid.org/0000-0003-3344-9579"]},"institutions":[{"id":"https://openalex.org/I74796645","display_name":"Birla Institute of Technology and Science, Pilani","ror":"https://ror.org/001p3jz28","country_code":"IN","type":"education","lineage":["https://openalex.org/I74796645"]}],"countries":["IN"],"is_corresponding":false,"raw_author_name":"Anupama Mittal","raw_affiliation_strings":["Department of Pharmacy, Birla Institute of Technology and Science, Pilani (BITS-Pilani), Vidya Vihar Campus, Pilani 333031, India"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Pharmacy, Birla Institute of Technology and Science, Pilani (BITS-Pilani), Vidya Vihar Campus, Pilani 333031, India","institution_ids":["https://openalex.org/I74796645"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5006813082","display_name":"Deepak Chitkara","orcid":"https://orcid.org/0000-0003-4174-7664","observed_orcids":["https://orcid.org/0000-0003-4174-7664"]},"institutions":[{"id":"https://openalex.org/I74796645","display_name":"Birla Institute of Technology and Science, Pilani","ror":"https://ror.org/001p3jz28","country_code":"IN","type":"education","lineage":["https://openalex.org/I74796645"]}],"countries":["IN"],"is_corresponding":true,"raw_author_name":"Deepak Chitkara","raw_affiliation_strings":["Department of Pharmacy, Birla Institute of Technology and Science, Pilani (BITS-Pilani), Vidya Vihar Campus, Pilani 333031, India"],"raw_orcid":"https://orcid.org/0000-0003-4174-7664","affiliations":[{"raw_affiliation_string":"Department of Pharmacy, Birla Institute of Technology and Science, Pilani (BITS-Pilani), Vidya Vihar Campus, Pilani 333031, India","institution_ids":["https://openalex.org/I74796645"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":2,"corresponding_author_ids":["https://openalex.org/A5006813082"],"corresponding_institution_ids":["https://openalex.org/I74796645"],"apc_list":{"value":0,"currency":"USD","value_usd":0},"apc_paid":null,"fwci":0.7561,"has_fulltext":true,"cited_by_count":32,"citation_normalized_percentile":{"value":0.62732095,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":{"min":95,"max":99},"biblio":{"volume":"17","issue":"2","first_page":"153","last_page":"176"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10725","display_name":"RNA Interference and Gene Delivery","score":0.5205000042915344,"subfield":{"id":"https://openalex.org/subfields/1312","display_name":"Molecular Biology"},"field":{"id":"https://openalex.org/fields/13","display_name":"Biochemistry, Genetics and Molecular Biology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},"topics":[{"id":"https://openalex.org/T10725","display_name":"RNA Interference and Gene Delivery","score":0.5205000042915344,"subfield":{"id":"https://openalex.org/subfields/1312","display_name":"Molecular Biology"},"field":{"id":"https://openalex.org/fields/13","display_name":"Biochemistry, Genetics and Molecular Biology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},{"id":"https://openalex.org/T10691","display_name":"Retinal Development and Disorders","score":0.3330000042915344,"subfield":{"id":"https://openalex.org/subfields/2731","display_name":"Ophthalmology"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}},{"id":"https://openalex.org/T10878","display_name":"CRISPR and Genetic Engineering","score":0.047200001776218414,"subfield":{"id":"https://openalex.org/subfields/1312","display_name":"Molecular Biology"},"field":{"id":"https://openalex.org/fields/13","display_name":"Biochemistry, Genetics and Molecular Biology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}}],"keywords":[{"id":"https://openalex.org/keywords/crispr-cas9","display_name":"CRISPR/Cas9","score":0.9150000214576721},{"id":"https://openalex.org/keywords/retinal-dystrophy","display_name":"retinal dystrophy","score":0.8970000147819519},{"id":"https://openalex.org/keywords/gene-delivery","display_name":"gene delivery","score":0.6340000033378601},{"id":"https://openalex.org/keywords/non-viral-vectors","display_name":"non-viral vectors","score":0.9049999713897705},{"id":"https://openalex.org/keywords/retinitis-pigmentosa","display_name":"retinitis pigmentosa","score":0.7879999876022339},{"id":"https://openalex.org/keywords/cone-rod-dystrophy","display_name":"cone-rod dystrophy","score":0.9129999876022339},{"id":"https://openalex.org/keywords/congenital-stationary-night-blindness","display_name":"congenital stationary night blindness","score":0.8080000281333923},{"id":"https://openalex.org/keywords/x-linked-retinoschisis","display_name":"X-linked retinoschisis","score":0.9010000228881836},{"id":"https://openalex.org/keywords/age-related-macular-degeneration","display_name":"age-related macular degeneration","score":0.9269999861717224},{"id":"https://openalex.org/keywords/leber-congenital-amaurosis","display_name":"Leber congenital amaurosis","score":0.9419999718666077},{"id":"https://openalex.org/keywords/genome-editing","display_name":"genome editing","score":0.44200000166893005}],"concepts":[{"id":"https://openalex.org/C98108389","wikidata":"https://www.wikidata.org/wiki/Q412563","display_name":"CRISPR","level":3,"score":0.8411327004432678},{"id":"https://openalex.org/C144501496","wikidata":"https://www.wikidata.org/wiki/Q5533489","display_name":"Genome editing","level":4,"score":0.6708521842956543},{"id":"https://openalex.org/C2781114197","wikidata":"https://www.wikidata.org/wiki/Q847057","display_name":"Retinitis pigmentosa","level":3,"score":0.6450362205505371},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.5573195219039917},{"id":"https://openalex.org/C2781040256","wikidata":"https://www.wikidata.org/wiki/Q7316761","display_name":"Retinal degeneration","level":3,"score":0.49042174220085144},{"id":"https://openalex.org/C2779460726","wikidata":"https://www.wikidata.org/wiki/Q21115224","display_name":"ABCA4","level":4,"score":0.483980655670166},{"id":"https://openalex.org/C2780827179","wikidata":"https://www.wikidata.org/wiki/Q422001","display_name":"Retinal","level":2,"score":0.48181405663490295},{"id":"https://openalex.org/C111599444","wikidata":"https://www.wikidata.org/wiki/Q213901","display_name":"Genetic enhancement","level":3,"score":0.4746552109718323},{"id":"https://openalex.org/C132455925","wikidata":"https://www.wikidata.org/wiki/Q16965677","display_name":"Cas9","level":4,"score":0.4128764271736145},{"id":"https://openalex.org/C54355233","wikidata":"https://www.wikidata.org/wiki/Q7162","display_name":"Genetics","level":1,"score":0.40292614698410034},{"id":"https://openalex.org/C70721500","wikidata":"https://www.wikidata.org/wiki/Q177005","display_name":"Computational biology","level":1,"score":0.34630101919174194},{"id":"https://openalex.org/C60644358","wikidata":"https://www.wikidata.org/wiki/Q128570","display_name":"Bioinformatics","level":1,"score":0.3370835781097412},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.28435879945755005},{"id":"https://openalex.org/C127716648","wikidata":"https://www.wikidata.org/wiki/Q104053","display_name":"Phenotype","level":3,"score":0.07597240805625916},{"id":"https://openalex.org/C55493867","wikidata":"https://www.wikidata.org/wiki/Q7094","display_name":"Biochemistry","level":1,"score":0.0}],"mesh":[],"locations_count":4,"locations":[{"id":"doi:10.1016/j.ajps.2022.02.001","doi":"https://doi.org/10.1016/j.ajps.2022.02.001","is_oa":true,"landing_page_url":"https://doi.org/10.1016/j.ajps.2022.02.001","pdf_url":"https://www.sciencedirect.com/science/article/pii/S1818087622000149/pdf","source":{"id":"https://openalex.org/S2764650913","display_name":"Asian Journal of Pharmaceutical Sciences","issn_l":"1818-0876","issn":["1818-0876","2221-285X"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["cwts-core","doaj","dongbi-c","doyens","jufo-1","ki-jl-1","oarl","poland-100","russia-white-list-1"],"host_organization":"https://openalex.org/P4310320990","host_organization_name":"Elsevier BV","host_organization_lineage":["https://openalex.org/P4310320990"],"host_organization_lineage_names":["Elsevier BV"],"type":"journal"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Asian Journal of Pharmaceutical Sciences","raw_type":"journal-article"},{"id":"pmh:oai:pubmedcentral.nih.gov:9614410","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/9614410","pdf_url":null,"source":{"id":"https://openalex.org/S2764455111","display_name":"PubMed Central","issn_l":null,"issn":null,"is_oa":true,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1299303238","host_organization_name":"National Institutes of Health","host_organization_lineage":["https://openalex.org/I1299303238"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Asian J Pharm Sci","raw_type":"Text"},{"id":"pmid:36320315","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/36320315","pdf_url":null,"source":{"id":"https://openalex.org/S4306525036","display_name":"PubMed","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1299303238","host_organization_name":"National Institutes of Health","host_organization_lineage":["https://openalex.org/I1299303238"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Asian journal of pharmaceutical sciences","raw_type":"Journal Article"},{"id":"pmh:oai:doaj.org/article:3ab14c82266e4a39ac11ddc10cdd9c17","doi":null,"is_oa":true,"landing_page_url":"https://doaj.org/article/3ab14c82266e4a39ac11ddc10cdd9c17","pdf_url":null,"source":{"id":"https://openalex.org/S4306401280","display_name":"DOAJ (DOAJ: Directory of Open Access Journals)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":null,"host_organization_name":null,"host_organization_lineage":[],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by-sa","license_id":"https://openalex.org/licenses/cc-by-sa","version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"Asian Journal of Pharmaceutical Sciences, Vol 17, Iss 2, Pp 153-176 (2022)","raw_type":"article"}],"best_oa_location":{"id":"doi:10.1016/j.ajps.2022.02.001","doi":"https://doi.org/10.1016/j.ajps.2022.02.001","is_oa":true,"landing_page_url":"https://doi.org/10.1016/j.ajps.2022.02.001","pdf_url":"https://www.sciencedirect.com/science/article/pii/S1818087622000149/pdf","source":{"id":"https://openalex.org/S2764650913","display_name":"Asian Journal of Pharmaceutical Sciences","issn_l":"1818-0876","issn":["1818-0876","2221-285X"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["cwts-core","doaj","dongbi-c","doyens","jufo-1","ki-jl-1","oarl","poland-100","russia-white-list-1"],"host_organization":"https://openalex.org/P4310320990","host_organization_name":"Elsevier BV","host_organization_lineage":["https://openalex.org/P4310320990"],"host_organization_lineage_names":["Elsevier BV"],"type":"journal"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Asian Journal of Pharmaceutical Sciences","raw_type":"journal-article"},"sustainable_development_goals":[{"id":"https://metadata.un.org/sdg/3","display_name":"Good health and well-being","score":0.9476000070571899}],"study_designs":[],"awards":[{"id":"https://openalex.org/G1513965152","display_name":null,"funder_award_id":"BT/PR26897/NNT/28/1489/2017","funder_id":"https://openalex.org/F4320320717","funder_display_name":"Department of Biotechnology, Ministry of Science and Technology, India"},{"id":"https://openalex.org/G4673130745","display_name":null,"funder_award_id":"3/1/3/JRF-2019/HRD","funder_id":"https://openalex.org/F4320320720","funder_display_name":"Indian Council of Medical Research"},{"id":"https://openalex.org/G6399908727","display_name":null,"funder_award_id":"3/1/3","funder_id":"https://openalex.org/F4320320720","funder_display_name":"Indian Council of Medical Research"},{"id":"https://openalex.org/G8489118136","display_name":null,"funder_award_id":"JRF-2019","funder_id":"https://openalex.org/F4320320720","funder_display_name":"Indian Council of Medical Research"}],"funders":[{"id":"https://openalex.org/F4320320717","display_name":"Department of Biotechnology, Ministry of Science and Technology, India","ror":"https://ror.org/03tjsyq23"},{"id":"https://openalex.org/F4320320720","display_name":"Indian Council of Medical Research","ror":"https://ror.org/0492wrx28"}],"has_content":{"pdf":true,"grobid_xml":true},"content_urls":{"pdf":"https://content.openalex.org/works/W4212945596.pdf","grobid_xml":"https://content.openalex.org/works/W4212945596.grobid-xml"},"referenced_works_count":185,"referenced_works":["https://openalex.org/W60441999","https://openalex.org/W71344925","https://openalex.org/W114318986","https://openalex.org/W265142245","https://openalex.org/W1487528435","https://openalex.org/W1527494734","https://openalex.org/W1541621322","https://openalex.org/W1563465785","https://openalex.org/W1595068158","https://openalex.org/W1705739557","https://openalex.org/W1710257910","https://openalex.org/W1931935484","https://openalex.org/W1962249777","https://openalex.org/W1968853259","https://openalex.org/W1971290197","https://openalex.org/W1978137600","https://openalex.org/W1989130241","https://openalex.org/W1989405237","https://openalex.org/W1991393782","https://openalex.org/W1993642022","https://openalex.org/W1993913137","https://openalex.org/W1994936258","https://openalex.org/W1995140705","https://openalex.org/W1995284319","https://openalex.org/W1995956587","https://openalex.org/W1998533469","https://openalex.org/W1998566510","https://openalex.org/W2004687695","https://openalex.org/W2004906535","https://openalex.org/W2005837470","https://openalex.org/W2007097455","https://openalex.org/W2008289270","https://openalex.org/W2011085652","https://openalex.org/W2017986205","https://openalex.org/W2023606370","https://openalex.org/W2024098907","https://openalex.org/W2027568368","https://openalex.org/W2028006420","https://openalex.org/W2031564035","https://openalex.org/W2032141669","https://openalex.org/W2032760352","https://openalex.org/W2035233312","https://openalex.org/W2039551908","https://openalex.org/W2040541933","https://openalex.org/W2041395551","https://openalex.org/W2042209155","https://openalex.org/W2050774731","https://openalex.org/W2054359043","https://openalex.org/W2056602419","https://openalex.org/W2058647478","https://openalex.org/W2060063409","https://openalex.org/W2064469627","https://openalex.org/W2064815984","https://openalex.org/W2070120687","https://openalex.org/W2070614858","https://openalex.org/W2073621589","https://openalex.org/W2075218366","https://openalex.org/W2077659966","https://openalex.org/W2093279717","https://openalex.org/W2093664801","https://openalex.org/W2096454490","https://openalex.org/W2101749257","https://openalex.org/W2103376319","https://openalex.org/W2103609218","https://openalex.org/W2105464583","https://openalex.org/W2107046588","https://openalex.org/W2119164964","https://openalex.org/W2121750285","https://openalex.org/W2123366092","https://openalex.org/W2126115955","https://openalex.org/W2137222303","https://openalex.org/W2140022328","https://openalex.org/W2141575938","https://openalex.org/W2149751339","https://openalex.org/W2151809142","https://openalex.org/W2159187058","https://openalex.org/W2160051211","https://openalex.org/W2160550697","https://openalex.org/W2161728843","https://openalex.org/W2163173465","https://openalex.org/W2163241233","https://openalex.org/W2164088504","https://openalex.org/W2171214255","https://openalex.org/W2171695814","https://openalex.org/W2176608563","https://openalex.org/W2176963049","https://openalex.org/W2188223075","https://openalex.org/W2199356015","https://openalex.org/W2215305044","https://openalex.org/W2224398381","https://openalex.org/W2264231196","https://openalex.org/W2270424748","https://openalex.org/W2284395998","https://openalex.org/W2292390328","https://openalex.org/W2324350546","https://openalex.org/W2401478799","https://openalex.org/W2409002252","https://openalex.org/W2414673300","https://openalex.org/W2415526085","https://openalex.org/W2461013690","https://openalex.org/W2508248319","https://openalex.org/W2508903105","https://openalex.org/W2511569157","https://openalex.org/W2519167750","https://openalex.org/W2539812081","https://openalex.org/W2555520932","https://openalex.org/W2579979462","https://openalex.org/W2581151188","https://openalex.org/W2588613595","https://openalex.org/W2590764628","https://openalex.org/W2593151357","https://openalex.org/W2617204887","https://openalex.org/W2620315774","https://openalex.org/W2620518508","https://openalex.org/W2735350095","https://openalex.org/W2737167849","https://openalex.org/W2745749640","https://openalex.org/W2759557314","https://openalex.org/W2763042654","https://openalex.org/W2765080153","https://openalex.org/W2766599608","https://openalex.org/W2770505353","https://openalex.org/W2779736613","https://openalex.org/W2783755946","https://openalex.org/W2788789448","https://openalex.org/W2789344364","https://openalex.org/W2792141284","https://openalex.org/W2800176137","https://openalex.org/W2800319097","https://openalex.org/W2802060782","https://openalex.org/W2802426789","https://openalex.org/W2802499741","https://openalex.org/W2802665497","https://openalex.org/W2802963551","https://openalex.org/W2803039783","https://openalex.org/W2804566227","https://openalex.org/W2807488219","https://openalex.org/W2885768217","https://openalex.org/W2887151730","https://openalex.org/W2890167612","https://openalex.org/W2890684434","https://openalex.org/W2896481637","https://openalex.org/W2897639305","https://openalex.org/W2898360779","https://openalex.org/W2903861262","https://openalex.org/W2906030729","https://openalex.org/W2910850705","https://openalex.org/W2912972275","https://openalex.org/W2945739890","https://openalex.org/W2946451464","https://openalex.org/W2951794613","https://openalex.org/W2971284229","https://openalex.org/W2971876988","https://openalex.org/W2972907673","https://openalex.org/W2981589839","https://openalex.org/W2982498219","https://openalex.org/W2989678989","https://openalex.org/W2991183086","https://openalex.org/W2997563572","https://openalex.org/W3001996117","https://openalex.org/W3004457224","https://openalex.org/W3004643344","https://openalex.org/W3011522762","https://openalex.org/W3017421630","https://openalex.org/W3020907828","https://openalex.org/W3035960414","https://openalex.org/W3037664753","https://openalex.org/W3038068331","https://openalex.org/W3038956601","https://openalex.org/W3044730717","https://openalex.org/W3047882216","https://openalex.org/W3091666079","https://openalex.org/W3106735065","https://openalex.org/W3112781161","https://openalex.org/W3128825549","https://openalex.org/W3140318909","https://openalex.org/W3164236944","https://openalex.org/W3165289507","https://openalex.org/W4206892629","https://openalex.org/W4210375322","https://openalex.org/W4211099883","https://openalex.org/W4245053291","https://openalex.org/W6637579592","https://openalex.org/W6754177085","https://openalex.org/W7073540871"],"related_works":["https://openalex.org/W4294169508","https://openalex.org/W4402969161","https://openalex.org/W7204831981","https://openalex.org/W3164545206","https://openalex.org/W4284701301","https://openalex.org/W4407305626","https://openalex.org/W3144118534","https://openalex.org/W2789344364","https://openalex.org/W4399163193","https://openalex.org/W4411049668"],"abstract_inverted_index":{"CRISPR/Cas,":[0],"an":[1,11],"adaptive":[2],"immune":[3],"system":[4],"in":[5,46,97,103,143,159],"bacteria,":[6],"has":[7,25,43,108],"been":[8,26],"adopted":[9],"as":[10],"efficient":[12],"and":[13,53,142],"precise":[14],"tool":[15],"for":[16,28,76,118],"site-specific":[17],"gene":[18,34,88,101],"editing":[19],"with":[20],"potential":[21,158],"therapeutic":[22],"opportunities.":[23],"It":[24,42],"explored":[27],"a":[29,132],"variety":[30],"of":[31,73,85,100,114,128,146,170],"applications,":[32],"including":[33,50],"modulation,":[35],"epigenome":[36],"editing,":[37,40],"diagnosis,":[38],"mRNA":[39],"etc.":[41,71],"found":[44],"applications":[45],"retinal":[47,77,119,171],"dystrophic":[48,78,104],"conditions":[49,79],"progressive":[51],"cone":[52],"cone-rod":[54],"dystrophies,":[55],"congenital":[56,69],"stationary":[57],"night":[58],"blindness,":[59],"X-linked":[60],"juvenile":[61],"retinoschisis,":[62],"retinitis":[63],"pigmentosa,":[64],"age-related":[65],"macular":[66],"degeneration,":[67],"leber's":[68],"amaurosis,":[70],"Most":[72],"the":[74,87,98,112,125,129,168],"therapies":[75],"work":[80],"by":[81],"regressing":[82],"symptoms":[83],"instead":[84],"reversing":[86],"mutations.":[89],"CRISPR/Cas9":[90],"through":[91],"indel":[92],"could":[93],"impart":[94],"beneficial":[95],"effects":[96],"reversal":[99],"mutations":[102],"conditions.":[105],"Recent":[106],"research":[107],"also":[109],"consolidated":[110],"on":[111],"approaches":[113],"using":[115,174],"CRISPR":[116,147],"systems":[117],"dystrophies":[120,172],"but":[121],"their":[122,157],"delivery":[123],"to":[124,136,156],"posterior":[126],"part":[127],"eye":[130],"is":[131],"major":[133],"concern":[134],"due":[135,155],"high":[137],"molecular":[138],"weight,":[139],"negative":[140],"charge,":[141],"vivo":[144],"stability":[145],"components.":[148],"Recently,":[149],"non-viral":[150],"vectors":[151],"have":[152],"gained":[153],"interest":[154],"tissue-specific":[160],"nucleic":[161],"acid":[162],"(miRNA/siRNA/CRISPR)":[163],"delivery.":[164],"This":[165],"review":[166],"highlights":[167],"opportunities":[169],"management":[173],"CRISPR/Cas":[175],"nanomedicine.":[176]},"counts_by_year":[{"year":2026,"cited_by_count":2},{"year":2025,"cited_by_count":9},{"year":2024,"cited_by_count":7},{"year":2023,"cited_by_count":10},{"year":2022,"cited_by_count":4}],"updated_date":"2026-11-04T10:54:05.112534","created_date":"2022-02-24T00:00:00"}