{"id":"https://openalex.org/W7225425849","doi":"https://doi.org/10.11648/j.mhs.20260204.12","title":"Gene Therapy and CRISPR Technologies: Revolutionizing Medicine","display_name":"Gene Therapy and CRISPR Technologies: Revolutionizing Medicine","publication_year":2026,"publication_date":"2026-11-06","ids":{"openalex":"https://openalex.org/W7225425849","doi":"https://doi.org/10.11648/j.mhs.20260204.12"},"language":"en","primary_location":{"id":"doi:10.11648/j.mhs.20260204.12","doi":"https://doi.org/10.11648/j.mhs.20260204.12","is_oa":true,"landing_page_url":"https://doi.org/10.11648/j.mhs.20260204.12","pdf_url":"http://article.sciencepg.com/pdf/j.mhs.20260204.12","source":{"id":"https://openalex.org/S7407053595","display_name":"Medicine and Health Sciences","issn_l":"3070-6300","issn":["3070-6300"],"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/P4310319272","host_organization_name":"Science Publishing Group","host_organization_lineage":["https://openalex.org/P4310319272"],"host_organization_lineage_names":["Science Publishing Group"],"type":"journal"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Medicine and Health Sciences","raw_type":"journal-article"},"volume":null,"type":"article","is_traditional":true,"indexed_in":["crossref"],"open_access":{"is_oa":true,"oa_status":"hybrid","oa_url":"http://article.sciencepg.com/pdf/j.mhs.20260204.12","any_repository_has_fulltext":false},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5161075069","display_name":"Tharushi Silva","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4400008902","display_name":"Alte University","ror":"https://ror.org/041bgem97","country_code":"GE","type":"education","lineage":["https://openalex.org/I4400008902"]}],"countries":["GE"],"is_corresponding":true,"raw_author_name":"Tharushi Silva","raw_affiliation_strings":["International Medical School, Alte University, Tbilisi, Georgia"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"International Medical School, Alte University, Tbilisi, Georgia","institution_ids":["https://openalex.org/I4400008902"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5161036521","display_name":"Siavash Chermahini","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210126868","display_name":"University of Georgia","ror":"https://ror.org/02bjhwk41","country_code":"GE","type":"education","lineage":["https://openalex.org/I4210126868"]}],"countries":["GE"],"is_corresponding":false,"raw_author_name":"Siavash Chermahini","raw_affiliation_strings":["School of Science and Technology, The University of Georgia, Tbilisi, Georgia"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"School of Science and Technology, The University of Georgia, Tbilisi, Georgia","institution_ids":["https://openalex.org/I4210126868"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5161020817","display_name":"Pranav Balasubramonian","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4400008902","display_name":"Alte University","ror":"https://ror.org/041bgem97","country_code":"GE","type":"education","lineage":["https://openalex.org/I4400008902"]}],"countries":["GE"],"is_corresponding":false,"raw_author_name":"Pranav Balasubramonian","raw_affiliation_strings":["International Medical School, Alte University, Tbilisi, Georgia"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"International Medical School, Alte University, Tbilisi, Georgia","institution_ids":["https://openalex.org/I4400008902"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5160999925","display_name":"Kulsum Alam","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4400008902","display_name":"Alte University","ror":"https://ror.org/041bgem97","country_code":"GE","type":"education","lineage":["https://openalex.org/I4400008902"]}],"countries":["GE"],"is_corresponding":false,"raw_author_name":"Kulsum Alam","raw_affiliation_strings":["International Medical School, Alte University, Tbilisi, Georgia"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"International Medical School, Alte University, Tbilisi, Georgia","institution_ids":["https://openalex.org/I4400008902"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5161011004","display_name":"Vedant Malekar","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4400008902","display_name":"Alte University","ror":"https://ror.org/041bgem97","country_code":"GE","type":"education","lineage":["https://openalex.org/I4400008902"]}],"countries":["GE"],"is_corresponding":false,"raw_author_name":"Vedant Malekar","raw_affiliation_strings":["International Medical School, Alte University, Tbilisi, Georgia"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"International Medical School, Alte University, Tbilisi, Georgia","institution_ids":["https://openalex.org/I4400008902"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5161108210","display_name":"Manthan Jain","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4400008902","display_name":"Alte University","ror":"https://ror.org/041bgem97","country_code":"GE","type":"education","lineage":["https://openalex.org/I4400008902"]}],"countries":["GE"],"is_corresponding":false,"raw_author_name":"Manthan Jain","raw_affiliation_strings":["International Medical School, Alte University, Tbilisi, Georgia"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"International Medical School, Alte University, Tbilisi, Georgia","institution_ids":["https://openalex.org/I4400008902"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5100982498","display_name":"Nishaat Ahmed","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4400008902","display_name":"Alte University","ror":"https://ror.org/041bgem97","country_code":"GE","type":"education","lineage":["https://openalex.org/I4400008902"]}],"countries":["GE"],"is_corresponding":false,"raw_author_name":"Nishaat Ahmed","raw_affiliation_strings":["International Medical School, Alte University, Tbilisi, Georgia"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"International Medical School, Alte University, Tbilisi, Georgia","institution_ids":["https://openalex.org/I4400008902"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":2,"corresponding_author_ids":["https://openalex.org/A5161075069"],"corresponding_institution_ids":["https://openalex.org/I4400008902"],"apc_list":null,"apc_paid":null,"fwci":0.0,"has_fulltext":true,"cited_by_count":0,"citation_normalized_percentile":{"value":0.0,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":null,"biblio":{"volume":"2","issue":"4","first_page":"128","last_page":"131"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10878","display_name":"CRISPR and Genetic Engineering","score":0.9907000064849854,"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/T10878","display_name":"CRISPR and Genetic Engineering","score":0.9907000064849854,"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/T10613","display_name":"Virus-based gene therapy research","score":0.007600000128149986,"subfield":{"id":"https://openalex.org/subfields/1311","display_name":"Genetics"},"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/T10725","display_name":"RNA Interference and Gene Delivery","score":0.0003000000142492354,"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/gene-therapy","display_name":"gene therapy","score":0.9990000128746033},{"id":"https://openalex.org/keywords/crispr-cas-systems","display_name":"CRISPR-Cas","score":0.8740000128746033},{"id":"https://openalex.org/keywords/genome-editing","display_name":"genome editing","score":0.9959999918937683},{"id":"https://openalex.org/keywords/base-editing","display_name":"base editing","score":0.9599999785423279},{"id":"https://openalex.org/keywords/prime-editing","display_name":"prime editing","score":1.0},{"id":"https://openalex.org/keywords/gene-delivery-vectors","display_name":"gene delivery vectors","score":0.703000009059906},{"id":"https://openalex.org/keywords/viral-vectors","display_name":"viral vectors","score":0.9200000166893005},{"id":"https://openalex.org/keywords/non-viral-delivery","display_name":"non-viral delivery","score":0.765999972820282},{"id":"https://openalex.org/keywords/clinical-trials","display_name":"clinical trials","score":0.8320000171661377},{"id":"https://openalex.org/keywords/inherited-blood-disorders","display_name":"inherited blood disorders","score":0.9549999833106995},{"id":"https://openalex.org/keywords/cancer-therapy","display_name":"cancer therapy","score":0.7670000195503235},{"id":"https://openalex.org/keywords/metabolic-disorders","display_name":"metabolic disorders","score":0.9909999966621399},{"id":"https://openalex.org/keywords/off-target-effects","display_name":"off-target effects","score":0.996999979019165},{"id":"https://openalex.org/keywords/ethics-and-regulation","display_name":"ethics and regulation","score":0.6069999933242798}],"concepts":[{"id":"https://openalex.org/C144501496","wikidata":"https://www.wikidata.org/wiki/Q5533489","display_name":"Genome editing","level":4,"score":0.8500000238418579},{"id":"https://openalex.org/C98108389","wikidata":"https://www.wikidata.org/wiki/Q412563","display_name":"CRISPR","level":3,"score":0.8393999934196472},{"id":"https://openalex.org/C111599444","wikidata":"https://www.wikidata.org/wiki/Q213901","display_name":"Genetic enhancement","level":3,"score":0.7197999954223633},{"id":"https://openalex.org/C70721500","wikidata":"https://www.wikidata.org/wiki/Q177005","display_name":"Computational biology","level":1,"score":0.5278000235557556},{"id":"https://openalex.org/C132455925","wikidata":"https://www.wikidata.org/wiki/Q16965677","display_name":"Cas9","level":4,"score":0.4779999852180481},{"id":"https://openalex.org/C535046627","wikidata":"https://www.wikidata.org/wiki/Q30612","display_name":"Clinical trial","level":2,"score":0.47780001163482666},{"id":"https://openalex.org/C163763905","wikidata":"https://www.wikidata.org/wiki/Q17075943","display_name":"Precision medicine","level":2,"score":0.46970000863075256},{"id":"https://openalex.org/C135983454","wikidata":"https://www.wikidata.org/wiki/Q2790163","display_name":"Gene delivery","level":4,"score":0.42089998722076416},{"id":"https://openalex.org/C2779974597","wikidata":"https://www.wikidata.org/wiki/Q28448986","display_name":"Clinical Practice","level":2,"score":0.41940000653266907},{"id":"https://openalex.org/C60644358","wikidata":"https://www.wikidata.org/wiki/Q128570","display_name":"Bioinformatics","level":1,"score":0.4163999855518341},{"id":"https://openalex.org/C141231307","wikidata":"https://www.wikidata.org/wiki/Q7020","display_name":"Genome","level":3,"score":0.40209999680519104},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.3970000147819519},{"id":"https://openalex.org/C32470452","wikidata":"https://www.wikidata.org/wiki/Q2298489","display_name":"Viral vector","level":4,"score":0.3711000084877014},{"id":"https://openalex.org/C3018928802","wikidata":"https://www.wikidata.org/wiki/Q83185","display_name":"Gene transfer","level":3,"score":0.3666999936103821},{"id":"https://openalex.org/C35802184","wikidata":"https://www.wikidata.org/wiki/Q204773","display_name":"Zinc finger nuclease","level":5,"score":0.3515999913215637},{"id":"https://openalex.org/C197077220","wikidata":"https://www.wikidata.org/wiki/Q720988","display_name":"Human genome","level":4,"score":0.34459999203681946},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.3353999853134155},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.32899999618530273},{"id":"https://openalex.org/C189206191","wikidata":"https://www.wikidata.org/wiki/Q222046","display_name":"Genomics","level":4,"score":0.3163999915122986},{"id":"https://openalex.org/C191908910","wikidata":"https://www.wikidata.org/wiki/Q862838","display_name":"Synthetic biology","level":2,"score":0.30809998512268066},{"id":"https://openalex.org/C41008148","wikidata":"https://www.wikidata.org/wiki/Q21198","display_name":"Computer science","level":0,"score":0.30390000343322754},{"id":"https://openalex.org/C32220436","wikidata":"https://www.wikidata.org/wiki/Q2072214","display_name":"Personalized medicine","level":2,"score":0.30239999294281006},{"id":"https://openalex.org/C66782513","wikidata":"https://www.wikidata.org/wiki/Q864601","display_name":"Biomedicine","level":2,"score":0.28760001063346863},{"id":"https://openalex.org/C145290725","wikidata":"https://www.wikidata.org/wiki/Q5533489","display_name":"Genome engineering","level":5,"score":0.2806999981403351},{"id":"https://openalex.org/C162264550","wikidata":"https://www.wikidata.org/wiki/Q17112037","display_name":"Translational medicine","level":2,"score":0.2791000008583069},{"id":"https://openalex.org/C2910788575","wikidata":"https://www.wikidata.org/wiki/Q200779","display_name":"Hereditary Diseases","level":3,"score":0.2741999924182892},{"id":"https://openalex.org/C23837897","wikidata":"https://www.wikidata.org/wiki/Q159236","display_name":"Genetic engineering","level":3,"score":0.27140000462532043},{"id":"https://openalex.org/C2986663376","wikidata":"https://www.wikidata.org/wiki/Q9465","display_name":"Ethical issues","level":2,"score":0.2689000070095062},{"id":"https://openalex.org/C8891405","wikidata":"https://www.wikidata.org/wiki/Q1059","display_name":"Immune system","level":2,"score":0.26669999957084656},{"id":"https://openalex.org/C112930515","wikidata":"https://www.wikidata.org/wiki/Q4389547","display_name":"Risk analysis (engineering)","level":1,"score":0.2660999894142151},{"id":"https://openalex.org/C2779134260","wikidata":"https://www.wikidata.org/wiki/Q12136","display_name":"Disease","level":2,"score":0.2551000118255615},{"id":"https://openalex.org/C110894328","wikidata":"https://www.wikidata.org/wiki/Q4086911","display_name":"Biomedical technology","level":2,"score":0.2538999915122986}],"mesh":[],"locations_count":1,"locations":[{"id":"doi:10.11648/j.mhs.20260204.12","doi":"https://doi.org/10.11648/j.mhs.20260204.12","is_oa":true,"landing_page_url":"https://doi.org/10.11648/j.mhs.20260204.12","pdf_url":"http://article.sciencepg.com/pdf/j.mhs.20260204.12","source":{"id":"https://openalex.org/S7407053595","display_name":"Medicine and Health Sciences","issn_l":"3070-6300","issn":["3070-6300"],"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/P4310319272","host_organization_name":"Science Publishing Group","host_organization_lineage":["https://openalex.org/P4310319272"],"host_organization_lineage_names":["Science Publishing Group"],"type":"journal"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Medicine and Health Sciences","raw_type":"journal-article"}],"best_oa_location":{"id":"doi:10.11648/j.mhs.20260204.12","doi":"https://doi.org/10.11648/j.mhs.20260204.12","is_oa":true,"landing_page_url":"https://doi.org/10.11648/j.mhs.20260204.12","pdf_url":"http://article.sciencepg.com/pdf/j.mhs.20260204.12","source":{"id":"https://openalex.org/S7407053595","display_name":"Medicine and Health Sciences","issn_l":"3070-6300","issn":["3070-6300"],"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/P4310319272","host_organization_name":"Science Publishing Group","host_organization_lineage":["https://openalex.org/P4310319272"],"host_organization_lineage_names":["Science Publishing Group"],"type":"journal"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Medicine and Health Sciences","raw_type":"journal-article"},"sustainable_development_goals":[{"id":"https://metadata.un.org/sdg/3","display_name":"Good health and well-being","score":0.8048999905586243}],"study_designs":[],"awards":[],"funders":[],"has_content":{"grobid_xml":true,"pdf":true},"content_urls":{"pdf":"https://content.openalex.org/works/W7225425849.pdf","grobid_xml":"https://content.openalex.org/works/W7225425849.grobid-xml"},"referenced_works_count":16,"referenced_works":["https://openalex.org/W2045435533","https://openalex.org/W2050480410","https://openalex.org/W2064815984","https://openalex.org/W2127862779","https://openalex.org/W3112179900","https://openalex.org/W4206052126","https://openalex.org/W4213070361","https://openalex.org/W4296407084","https://openalex.org/W4390561922","https://openalex.org/W4390680704","https://openalex.org/W4394722849","https://openalex.org/W4396720445","https://openalex.org/W4399608254","https://openalex.org/W4406797974","https://openalex.org/W4412491129","https://openalex.org/W4416581309"],"related_works":["https://openalex.org/W4408994282","https://openalex.org/W7084589637","https://openalex.org/W4407785021","https://openalex.org/W7225114697","https://openalex.org/W4410309149","https://openalex.org/W7221787181","https://openalex.org/W7153216389","https://openalex.org/W4413767393","https://openalex.org/W7213918531","https://openalex.org/W4417535790"],"abstract_inverted_index":{"Gene":[0],"therapy":[1,75],"and":[2,19,58,76,88,97,105,116,146,154,163,173,182,203,229],"genome":[3,108,168],"editing":[4,96,99,169],"technologies":[5,187],"have":[6,52,100,120,134],"revolutionized":[7],"modern":[8],"medicine":[9,220],"by":[10,40],"allowing":[11],"for":[12,23,178],"the":[13,70,103,122,139,180,195,213,217],"precise":[14],"correction":[15],"of":[16,107,124,141,151,185,215,219],"genetic":[17],"defects":[18],"novel":[20],"therapeutic":[21,83,125],"approaches":[22],"diseases":[24,39],"that":[25,34,193],"were":[26],"previously":[27],"considered":[28],"untreatable.":[29],"It":[30],"is":[31,157],"a":[32],"technique":[33],"treats":[35],"or":[36,43],"prevents":[37],"various":[38],"replacing,":[41],"correcting":[42],"inactivating":[44],"faulty":[45],"genes.":[46],"Among":[47],"these":[48,186],"technologies,":[49,78],"CRISPR-Cas":[50],"systems":[51,119],"emerged":[53],"as":[54,94],"particularly":[55],"simple,":[56],"flexible,":[57],"rapidly":[59],"expanding":[60],"in":[61,73,114,138,167,188],"their":[62],"clinical":[63,85,132,189],"applications.":[64,170],"This":[65],"review":[66],"aims":[67],"to":[68,207,225],"synthesize":[69],"latest":[71],"advances":[72,113],"gene":[74,126],"CRISPR":[77],"including":[79],"mechanisms,":[80],"delivery":[81,118],"systems,":[82],"applications,":[84],"trials,":[86],"challenges,":[87],"future":[89],"perspectives.":[90],"Recent":[91],"developments":[92],"such":[93],"base":[95],"prime":[98],"significantly":[101],"improved":[102],"accuracy":[104],"safety":[106,201],"modification":[109],"approaches.":[110],"In":[111],"addition,":[112],"viral":[115],"non-viral":[117],"enhanced":[121],"efficiency":[123],"transfer":[127],"into":[128],"target":[129],"tissues.":[130],"Current":[131],"trials":[133],"demonstrated":[135],"promising":[136],"outcomes":[137],"treatment":[140],"inherited":[142],"blood":[143],"disorders,":[144],"cancers,":[145],"metabolic":[147],"diseases.":[148,231],"The":[149],"integration":[150],"artificial":[152],"intelligence":[153],"bioinformatics":[155],"tools":[156],"also":[158],"improving":[159],"guided":[160],"RNA":[161],"design":[162],"minimizing":[164],"off-target":[165],"effects":[166],"Ethical":[171],"considerations":[172],"regulatory":[174],"frameworks":[175],"remain":[176],"essential":[177],"ensuring":[179],"safe":[181],"equitable":[183],"implementation":[184],"practice.":[190],"We":[191],"conclude":[192],"despite":[194],"hurdles,":[196],"high":[197],"costs,":[198],"long":[199],"term":[200],"monitoring,":[202],"immune":[204],"system":[205],"reactions":[206],"vectors,":[208],"CRISPR-based":[209],"strategies":[210],"are":[211],"on":[212],"verge":[214],"revolutionizing":[216],"practice":[218],"with":[221],"innovative,":[222],"accurate":[223],"solutions":[224],"hard":[226],"hereditary,":[227],"oncologic,":[228],"infectious":[230]},"counts_by_year":[],"updated_date":"2026-11-08T12:12:28.789561","created_date":"2026-11-07T00:00:00"}