{"id":"https://openalex.org/W4383740461","doi":"https://doi.org/10.1002/cpt.3000","title":"Clinical Pharmacology and Translational Considerations in the Development of CRISPR\u2010Based Therapies","display_name":"Clinical Pharmacology and Translational Considerations in the Development of CRISPR\u2010Based Therapies","publication_year":2023,"publication_date":"2023-07-11","ids":{"openalex":"https://openalex.org/W4383740461","doi":"https://doi.org/10.1002/cpt.3000","pmid":"https://pubmed.ncbi.nlm.nih.gov/37429825"},"language":"en","primary_location":{"id":"doi:10.1002/cpt.3000","doi":"https://doi.org/10.1002/cpt.3000","is_oa":true,"landing_page_url":"https://doi.org/10.1002/cpt.3000","pdf_url":"https://onlinelibrary.wiley.com/doi/pdfdirect/10.1002/cpt.3000","source":{"id":"https://openalex.org/S159852663","display_name":"Clinical Pharmacology & Therapeutics","issn_l":"0009-9236","issn":["0009-9236","1085-8733","1532-6535"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-a","doyens","jufo-3","ki-jl-2","medline","norway-2","poland-140","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320595","host_organization_name":"Wiley","host_organization_lineage":["https://openalex.org/P4310320595"],"host_organization_lineage_names":["Wiley"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Clinical Pharmacology & Therapeutics","raw_type":"journal-article"},"volume":null,"type":"review","is_traditional":true,"indexed_in":["crossref","pubmed"],"open_access":{"is_oa":true,"oa_status":"bronze","oa_url":"https://onlinelibrary.wiley.com/doi/pdfdirect/10.1002/cpt.3000","any_repository_has_fulltext":false},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5102018126","display_name":"Ahmed M. Abdelhady","orcid":"https://orcid.org/0000-0001-9404-5397","observed_orcids":["https://orcid.org/0000-0001-9404-5397"]},"institutions":[{"id":"https://openalex.org/I4210131291","display_name":"Intellia Therapeutics (United States)","ror":"https://ror.org/03b368668","country_code":"US","type":"company","lineage":["https://openalex.org/I4210131291"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Ahmed M. Abdelhady","raw_affiliation_strings":["Intellia Therapeutics, Inc., Cambridge, Massachusetts, USA"],"raw_orcid":"https://orcid.org/0000-0001-9404-5397","affiliations":[{"raw_affiliation_string":"Intellia Therapeutics, Inc., Cambridge, Massachusetts, USA","institution_ids":["https://openalex.org/I4210131291"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5013190744","display_name":"Jonathan A. Phillips","orcid":"https://orcid.org/0000-0001-7290-754X","observed_orcids":["https://orcid.org/0000-0001-7290-754X"]},"institutions":[{"id":"https://openalex.org/I4210131291","display_name":"Intellia Therapeutics (United States)","ror":"https://ror.org/03b368668","country_code":"US","type":"company","lineage":["https://openalex.org/I4210131291"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Jonathan A. Phillips","raw_affiliation_strings":["Intellia Therapeutics, Inc., Cambridge, Massachusetts, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Intellia Therapeutics, Inc., Cambridge, Massachusetts, USA","institution_ids":["https://openalex.org/I4210131291"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5048689879","display_name":"Yuanxin Xu","orcid":"https://orcid.org/0000-0002-4878-1294","observed_orcids":["https://orcid.org/0000-0002-4878-1294"]},"institutions":[{"id":"https://openalex.org/I4210131291","display_name":"Intellia Therapeutics (United States)","ror":"https://ror.org/03b368668","country_code":"US","type":"company","lineage":["https://openalex.org/I4210131291"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Yuanxin Xu","raw_affiliation_strings":["Intellia Therapeutics, Inc., Cambridge, Massachusetts, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Intellia Therapeutics, Inc., Cambridge, Massachusetts, USA","institution_ids":["https://openalex.org/I4210131291"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5062831066","display_name":"Mark Stroh","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210131291","display_name":"Intellia Therapeutics (United States)","ror":"https://ror.org/03b368668","country_code":"US","type":"company","lineage":["https://openalex.org/I4210131291"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Mark Stroh","raw_affiliation_strings":["Intellia Therapeutics, Inc., Cambridge, Massachusetts, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Intellia Therapeutics, Inc., Cambridge, Massachusetts, USA","institution_ids":["https://openalex.org/I4210131291"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":1,"corresponding_author_ids":["https://openalex.org/A5062831066"],"corresponding_institution_ids":["https://openalex.org/I4210131291"],"apc_list":{"value":4000,"currency":"USD","value_usd":4000},"apc_paid":null,"fwci":0.1889,"has_fulltext":true,"cited_by_count":8,"citation_normalized_percentile":{"value":0.14450783,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":{"min":87,"max":97},"biblio":{"volume":"114","issue":"3","first_page":"591","last_page":"603"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10878","display_name":"CRISPR and Genetic Engineering","score":0.9771000146865845,"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":0.9771000146865845,"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":0.006399999838322401,"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":0.002199999988079071,"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-based-therapy","display_name":"CRISPR-based therapy","score":0.9020000100135803},{"id":"https://openalex.org/keywords/genome-editing","display_name":"genome editing","score":0.9649999737739563},{"id":"https://openalex.org/keywords/clinical-pharmacology","display_name":"clinical pharmacology","score":0.949999988079071},{"id":"https://openalex.org/keywords/translational-medicine","display_name":"translational medicine","score":0.7160000205039978},{"id":"https://openalex.org/keywords/pharmacokinetics","display_name":"pharmacokinetics","score":0.8240000009536743},{"id":"https://openalex.org/keywords/pharmacodynamics","display_name":"pharmacodynamics","score":0.9909999966621399},{"id":"https://openalex.org/keywords/immunogenicity","display_name":"immunogenicity","score":0.9319999814033508},{"id":"https://openalex.org/keywords/model-informed-drug-development","display_name":"model-informed drug development","score":0.8220000267028809},{"id":"https://openalex.org/keywords/dose-selection","display_name":"dose selection","score":0.8790000081062317},{"id":"https://openalex.org/keywords/ex-vivo-gene-therapy","display_name":"ex vivo gene therapy","score":0.6230000257492065},{"id":"https://openalex.org/keywords/in-vivo-gene-therapy","display_name":"in vivo gene therapy","score":0.875}],"concepts":[{"id":"https://openalex.org/C98108389","wikidata":"https://www.wikidata.org/wiki/Q412563","display_name":"CRISPR","level":3,"score":0.9287142157554626},{"id":"https://openalex.org/C144501496","wikidata":"https://www.wikidata.org/wiki/Q5533489","display_name":"Genome editing","level":4,"score":0.6380324363708496},{"id":"https://openalex.org/C70721500","wikidata":"https://www.wikidata.org/wiki/Q177005","display_name":"Computational biology","level":1,"score":0.5818656086921692},{"id":"https://openalex.org/C3020000666","wikidata":"https://www.wikidata.org/wiki/Q17112037","display_name":"Bench to bedside","level":2,"score":0.5252323150634766},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.4730709493160248},{"id":"https://openalex.org/C162264550","wikidata":"https://www.wikidata.org/wiki/Q17112037","display_name":"Translational medicine","level":2,"score":0.4396606683731079},{"id":"https://openalex.org/C128544194","wikidata":"https://www.wikidata.org/wiki/Q474859","display_name":"Translational research","level":2,"score":0.428432822227478},{"id":"https://openalex.org/C63433958","wikidata":"https://www.wikidata.org/wiki/Q1774688","display_name":"Clinical pharmacology","level":2,"score":0.42456015944480896},{"id":"https://openalex.org/C60644358","wikidata":"https://www.wikidata.org/wiki/Q128570","display_name":"Bioinformatics","level":1,"score":0.32973432540893555},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.2697732746601105},{"id":"https://openalex.org/C98274493","wikidata":"https://www.wikidata.org/wiki/Q128406","display_name":"Pharmacology","level":1,"score":0.25905299186706543},{"id":"https://openalex.org/C54355233","wikidata":"https://www.wikidata.org/wiki/Q7162","display_name":"Genetics","level":1,"score":0.1748608648777008},{"id":"https://openalex.org/C19527891","wikidata":"https://www.wikidata.org/wiki/Q1120908","display_name":"Medical physics","level":1,"score":0.09817984700202942},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.0858231782913208},{"id":"https://openalex.org/C142724271","wikidata":"https://www.wikidata.org/wiki/Q7208","display_name":"Pathology","level":1,"score":0.0}],"mesh":[{"descriptor_ui":"D000072669","descriptor_name":"Gene Editing","qualifier_ui":"Q000379","qualifier_name":"methods","is_major_topic":false},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D010601","descriptor_name":"Pharmacology, Clinical","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D064113","descriptor_name":"CRISPR-Cas Systems","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":true}],"locations_count":2,"locations":[{"id":"doi:10.1002/cpt.3000","doi":"https://doi.org/10.1002/cpt.3000","is_oa":true,"landing_page_url":"https://doi.org/10.1002/cpt.3000","pdf_url":"https://onlinelibrary.wiley.com/doi/pdfdirect/10.1002/cpt.3000","source":{"id":"https://openalex.org/S159852663","display_name":"Clinical Pharmacology & Therapeutics","issn_l":"0009-9236","issn":["0009-9236","1085-8733","1532-6535"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-a","doyens","jufo-3","ki-jl-2","medline","norway-2","poland-140","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320595","host_organization_name":"Wiley","host_organization_lineage":["https://openalex.org/P4310320595"],"host_organization_lineage_names":["Wiley"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Clinical Pharmacology & Therapeutics","raw_type":"journal-article"},{"id":"pmid:37429825","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/37429825","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":"Clinical pharmacology and therapeutics","raw_type":"Journal Article"}],"best_oa_location":{"id":"doi:10.1002/cpt.3000","doi":"https://doi.org/10.1002/cpt.3000","is_oa":true,"landing_page_url":"https://doi.org/10.1002/cpt.3000","pdf_url":"https://onlinelibrary.wiley.com/doi/pdfdirect/10.1002/cpt.3000","source":{"id":"https://openalex.org/S159852663","display_name":"Clinical Pharmacology & Therapeutics","issn_l":"0009-9236","issn":["0009-9236","1085-8733","1532-6535"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-a","doyens","jufo-3","ki-jl-2","medline","norway-2","poland-140","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320595","host_organization_name":"Wiley","host_organization_lineage":["https://openalex.org/P4310320595"],"host_organization_lineage_names":["Wiley"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Clinical Pharmacology & Therapeutics","raw_type":"journal-article"},"sustainable_development_goals":[{"score":0.9391000270843506,"id":"https://metadata.un.org/sdg/3","display_name":"Good health and well-being"}],"study_designs":[],"awards":[],"funders":[],"has_content":{"grobid_xml":true,"pdf":true},"content_urls":{"pdf":"https://content.openalex.org/works/W4383740461.pdf","grobid_xml":"https://content.openalex.org/works/W4383740461.grobid-xml"},"referenced_works_count":81,"referenced_works":["https://openalex.org/W149315227","https://openalex.org/W1545841075","https://openalex.org/W1545850783","https://openalex.org/W1965097716","https://openalex.org/W1968583232","https://openalex.org/W1976329054","https://openalex.org/W1994064996","https://openalex.org/W2003797386","https://openalex.org/W2006373538","https://openalex.org/W2032583774","https://openalex.org/W2045435533","https://openalex.org/W2046160219","https://openalex.org/W2058800908","https://openalex.org/W2064815984","https://openalex.org/W2115603949","https://openalex.org/W2118796952","https://openalex.org/W2129702476","https://openalex.org/W2143743183","https://openalex.org/W2144491853","https://openalex.org/W2152569432","https://openalex.org/W2161958391","https://openalex.org/W2164545730","https://openalex.org/W2167550136","https://openalex.org/W2168097562","https://openalex.org/W2199765595","https://openalex.org/W2233414233","https://openalex.org/W2247762492","https://openalex.org/W2253376971","https://openalex.org/W2262286634","https://openalex.org/W2336637054","https://openalex.org/W2339327563","https://openalex.org/W2342423058","https://openalex.org/W2519710256","https://openalex.org/W2548694234","https://openalex.org/W2594924162","https://openalex.org/W2611134270","https://openalex.org/W2658137558","https://openalex.org/W2760653955","https://openalex.org/W2784126913","https://openalex.org/W2787301955","https://openalex.org/W2788997515","https://openalex.org/W2891902322","https://openalex.org/W2900391822","https://openalex.org/W2901124589","https://openalex.org/W2903444830","https://openalex.org/W2921866483","https://openalex.org/W2939552562","https://openalex.org/W2972390440","https://openalex.org/W2990014136","https://openalex.org/W2998345757","https://openalex.org/W3002082131","https://openalex.org/W3005529704","https://openalex.org/W3006233208","https://openalex.org/W3011895208","https://openalex.org/W3017464623","https://openalex.org/W3039467801","https://openalex.org/W3040633579","https://openalex.org/W3044350523","https://openalex.org/W3120017373","https://openalex.org/W3126231081","https://openalex.org/W3130224201","https://openalex.org/W3130440716","https://openalex.org/W3134845766","https://openalex.org/W3155322721","https://openalex.org/W3171217459","https://openalex.org/W3177445623","https://openalex.org/W3183170971","https://openalex.org/W3207886513","https://openalex.org/W4200255906","https://openalex.org/W4206974976","https://openalex.org/W4210282011","https://openalex.org/W4213164504","https://openalex.org/W4283778388","https://openalex.org/W4283804613","https://openalex.org/W4285076926","https://openalex.org/W4293724129","https://openalex.org/W4308903662","https://openalex.org/W4309264705","https://openalex.org/W4311209630","https://openalex.org/W4317436849","https://openalex.org/W6737034060"],"related_works":["https://openalex.org/W4396850506","https://openalex.org/W7214975951","https://openalex.org/W2995257718","https://openalex.org/W3202725292","https://openalex.org/W3081375952","https://openalex.org/W4402694054","https://openalex.org/W3041816032","https://openalex.org/W7211944189","https://openalex.org/W4200255906","https://openalex.org/W7220742491"],"abstract_inverted_index":{"Genome":[0],"editing":[1,51],"holds":[2],"the":[3,29,46,53,66,84,88,106,120,132,147,177,196,205,233,247],"potential":[4],"for":[5,49,105,151,158,214],"curative":[6],"treatments":[7],"of":[8,57,68,75,91,109,122,146,149,169,176,179,186,199,222,235],"human":[9],"disease,":[10],"however,":[11],"clinical":[12,79,159,187,203,226],"realization":[13],"has":[14,93,100,231],"proven":[15],"to":[16,63,87,127,131,208],"be":[17],"a":[18,140,220],"challenging":[19],"journey":[20],"with":[21,78,139,211],"incremental":[22],"progress":[23,56],"made":[24],"up":[25],"until":[26],"recently.":[27],"Over":[28],"last":[30],"decade,":[31],"advances":[32,70,165],"in":[33,52,72,102,155,166,202,225,238,246],"clustered":[34],"regularly":[35],"interspaced":[36],"short":[37],"palindromic":[38],"repeats":[39],"(CRISPR)/CRISPR-associated":[40],"protein":[41],"(Cas)":[42],"systems":[43],"have":[44,172,182],"provided":[45],"necessary":[47],"breakthrough":[48],"genome":[50,133],"clinic.":[54,248],"The":[55],"investigational":[58,244],"CRISPR":[59,85,124,152,170,200],"therapies":[60,125,153,171,201,245],"from":[61,191],"bench":[62],"bedside":[64],"reflects":[65],"culmination":[67],"multiple":[69],"occurring":[71],"parallel,":[73],"several":[74],"which":[76],"intersect":[77],"pharmacology":[80,227],"and":[81,98,112,134,161,181,189,228,240],"translation.":[82],"Directing":[83],"therapy":[86],"intended":[89],"site":[90,121],"action":[92,150,180],"necessitated":[94],"novel":[95],"delivery":[96],"platforms,":[97],"this":[99],"resulted":[101],"special":[103],"considerations":[104,157],"complete":[107],"characterization":[108],"distribution,":[110],"metabolism,":[111],"excretion,":[113],"as":[114,116],"well":[115],"immunogenicity.":[117],"Once":[118],"at":[119],"action,":[123],"aim":[126],"make":[128],"permanent":[129],"alterations":[130],"achieve":[135],"therapeutically":[136],"relevant":[137],"effects":[138],"single":[141],"dose.":[142],"This":[143],"fundamental":[144],"aspect":[145],"mechanism":[148,178],"results":[154],"new":[156],"translation":[160,229],"dose":[162],"selection.":[163],"Early":[164],"model-informed":[167],"development":[168],"incorporated":[173],"key":[174],"facets":[175],"captured":[183],"hallmark":[184],"features":[185],"pharmacokinetics":[188],"pharmacodynamics":[190],"phase":[192],"I":[193],"investigations.":[194],"Given":[195],"recent":[197],"emergence":[198],"development,":[204],"landscape":[206],"continues":[207],"evolve":[209],"rapidly":[210],"ample":[212],"opportunity":[213],"continued":[215],"innovation.":[216],"Here,":[217],"we":[218],"provide":[219],"snapshot":[221],"selected":[223],"topics":[224],"that":[230],"supported":[232],"advance":[234],"systemically":[236],"administered":[237],"vivo":[239,242],"ex":[241],"CRISPR-based":[243]},"counts_by_year":[{"year":2026,"cited_by_count":3},{"year":2025,"cited_by_count":2},{"year":2024,"cited_by_count":2},{"year":2023,"cited_by_count":1}],"updated_date":"2026-11-12T12:28:57.059296","created_date":"2023-07-11T00:00:00"}