{"id":"https://openalex.org/W960773756","doi":null,"title":"MDM2 Targeting sd-rxRNA\u00ae for Retinoblastoma Therapy","display_name":"MDM2 Targeting sd-rxRNA\u00ae for Retinoblastoma Therapy","publication_year":2015,"publication_date":"2015-06-11","ids":{"openalex":"https://openalex.org/W960773756","mag":"960773756"},"language":"en","primary_location":{"id":"mag:960773756","doi":null,"is_oa":false,"landing_page_url":"http://investors.rxipharma.com/file/4418218/Index?KeyFile=1500078826","pdf_url":null,"source":{"id":"https://openalex.org/S76304953","display_name":"Investigative Ophthalmology & Visual Science","issn_l":"0146-0404","issn":["0146-0404","1552-5783"],"is_oa":false,"is_in_doaj":true,"is_core":true,"listed_in":["anvur-scientific","cwts-core","doaj","dongbi-c","doyens","jufo-2","ki-jl-2","medline","norway-2","oarl","poland-140","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310321950","host_organization_name":"Cadmus Press","host_organization_lineage":["https://openalex.org/P4310321950"],"host_organization_lineage_names":["Cadmus Press"],"type":"journal"},"license":null,"license_id":null,"version":null,"is_accepted":false,"is_published":false,"raw_source_name":"Investigative Ophthalmology & Visual Science","raw_type":null},"volume":null,"type":"article","is_traditional":true,"indexed_in":[],"open_access":{"is_oa":false,"oa_status":"closed","oa_url":null,"any_repository_has_fulltext":false},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5061526603","display_name":"Kevin Stachelek","orcid":"https://orcid.org/0000-0003-2085-695X","observed_orcids":["https://orcid.org/0000-0003-2085-695X"]},"institutions":[{"id":"https://openalex.org/I1336910626","display_name":"Children's Hospital Los Angeles","ror":"https://ror.org/00412ts95","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1336910626"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Kevin Stachelek","raw_affiliation_strings":["The Vision Center, Division of Ophthalmology, Children's Hospital Los Angeles, Los Angeles, CA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"The Vision Center, Division of Ophthalmology, Children's Hospital Los Angeles, Los Angeles, CA","institution_ids":["https://openalex.org/I1336910626"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5081951424","display_name":"James P. Cardia","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210123495","display_name":"Phio Pharmaceuticals (United States)","ror":"https://ror.org/034kn0n30","country_code":"US","type":"company","lineage":["https://openalex.org/I4210123495"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"James Cardia","raw_affiliation_strings":["RXi Pharmaceuticals, Marlborough, MA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"RXi Pharmaceuticals, Marlborough, MA","institution_ids":["https://openalex.org/I4210123495"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5052249996","display_name":"Dong-Lai Qi","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I1336910626","display_name":"Children's Hospital Los Angeles","ror":"https://ror.org/00412ts95","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1336910626"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Dong-Lai Qi","raw_affiliation_strings":["The Vision Center, Division of Ophthalmology, Children's Hospital Los Angeles, Los Angeles, CA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"The Vision Center, Division of Ophthalmology, Children's Hospital Los Angeles, Los Angeles, CA","institution_ids":["https://openalex.org/I1336910626"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5047473395","display_name":"Hardeep Pal Singh","orcid":"https://orcid.org/0000-0002-9586-8544","observed_orcids":["https://orcid.org/0000-0002-9586-8544"]},"institutions":[{"id":"https://openalex.org/I1336910626","display_name":"Children's Hospital Los Angeles","ror":"https://ror.org/00412ts95","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1336910626"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Hardeep Singh","raw_affiliation_strings":["The Vision Center, Division of Ophthalmology, Children's Hospital Los Angeles, Los Angeles, CA"],"raw_orcid":"https://orcid.org/0000-0002-9586-8544","affiliations":[{"raw_affiliation_string":"The Vision Center, Division of Ophthalmology, Children's Hospital Los Angeles, Los Angeles, CA","institution_ids":["https://openalex.org/I1336910626"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5046719934","display_name":"Lakshmipathi Pandarinathan","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210123495","display_name":"Phio Pharmaceuticals (United States)","ror":"https://ror.org/034kn0n30","country_code":"US","type":"company","lineage":["https://openalex.org/I4210123495"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Lakshmipathi Pandarinathan","raw_affiliation_strings":["RXi Pharmaceuticals, Marlborough, MA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"RXi Pharmaceuticals, Marlborough, MA","institution_ids":["https://openalex.org/I4210123495"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5003550993","display_name":"Katherine Holton","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210123495","display_name":"Phio Pharmaceuticals (United States)","ror":"https://ror.org/034kn0n30","country_code":"US","type":"company","lineage":["https://openalex.org/I4210123495"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Katherine Holton","raw_affiliation_strings":["RXi Pharmaceuticals, Marlborough, MA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"RXi Pharmaceuticals, Marlborough, MA","institution_ids":["https://openalex.org/I4210123495"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5053581595","display_name":"Karen G. Bulock","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210123495","display_name":"Phio Pharmaceuticals (United States)","ror":"https://ror.org/034kn0n30","country_code":"US","type":"company","lineage":["https://openalex.org/I4210123495"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Karen Bulock","raw_affiliation_strings":["RXi Pharmaceuticals, Marlborough, MA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"RXi Pharmaceuticals, Marlborough, MA","institution_ids":["https://openalex.org/I4210123495"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5083487972","display_name":"Pamela A. Pavco","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210123495","display_name":"Phio Pharmaceuticals (United States)","ror":"https://ror.org/034kn0n30","country_code":"US","type":"company","lineage":["https://openalex.org/I4210123495"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"P A Pavco","raw_affiliation_strings":["RXi Pharmaceuticals, Marlborough, MA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"RXi Pharmaceuticals, Marlborough, MA","institution_ids":["https://openalex.org/I4210123495"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5055676893","display_name":"David E. Cobrinik","orcid":"https://orcid.org/0000-0002-4478-2417","observed_orcids":["https://orcid.org/0000-0002-4478-2417"]},"institutions":[{"id":"https://openalex.org/I1174212","display_name":"University of Southern California","ror":"https://ror.org/03taz7m60","country_code":"US","type":"education","lineage":["https://openalex.org/I1174212"]},{"id":"https://openalex.org/I1336910626","display_name":"Children's Hospital Los Angeles","ror":"https://ror.org/00412ts95","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1336910626"]},{"id":"https://openalex.org/I4405270452","display_name":"Saban Research Institute","ror":"https://ror.org/00qe7sv26","country_code":"US","type":"facility","lineage":["https://openalex.org/I4405270452"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"David Cobrinik","raw_affiliation_strings":["USC Eye Institute Department of Ophthalmology, Keck School of Medicine, University of Southern California, Los Angeles, CA","The Vision Center, Division of Ophthalmology and the Saban Research Institute, Children's Hospital Los Angeles, Los Angeles, CA"],"raw_orcid":"https://orcid.org/0000-0002-4478-2417","affiliations":[{"raw_affiliation_string":"USC Eye Institute Department of Ophthalmology, Keck School of Medicine, University of Southern California, Los Angeles, CA","institution_ids":["https://openalex.org/I1174212"]},{"raw_affiliation_string":"The Vision Center, Division of Ophthalmology and the Saban Research Institute, Children's Hospital Los Angeles, Los Angeles, CA","institution_ids":["https://openalex.org/I1336910626","https://openalex.org/I4405270452"]}]},{"author_position":"last","author":{"id":null,"display_name":"Michael Byrne","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210123495","display_name":"Phio Pharmaceuticals (United States)","ror":"https://ror.org/034kn0n30","country_code":"US","type":"company","lineage":["https://openalex.org/I4210123495"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Michael Byrne","raw_affiliation_strings":["RXi Pharmaceuticals, Marlborough, MA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"RXi Pharmaceuticals, Marlborough, MA","institution_ids":["https://openalex.org/I4210123495"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":4,"corresponding_author_ids":[],"corresponding_institution_ids":[],"apc_list":{"value":1850,"currency":"USD","value_usd":1850},"apc_paid":null,"fwci":0.0,"has_fulltext":false,"cited_by_count":0,"citation_normalized_percentile":{"value":0.01218433,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":null,"biblio":{"volume":"56","issue":"7","first_page":"5473","last_page":"5473"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10725","display_name":"RNA Interference and Gene Delivery","score":0.6563000082969666,"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":0.6563000082969666,"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/T11328","display_name":"Ocular Oncology and Treatments","score":0.19539999961853027,"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/T10583","display_name":"Cancer-related Molecular Pathways","score":0.06650000065565109,"subfield":{"id":"https://openalex.org/subfields/2730","display_name":"Oncology"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}}],"keywords":[{"id":"https://openalex.org/keywords/mdm2","display_name":"MDM2","score":0.7490000128746033},{"id":"https://openalex.org/keywords/retinoblastoma","display_name":"retinoblastoma","score":0.9729999899864197},{"id":"https://openalex.org/keywords/mycn","display_name":"MYCN","score":0.7820000052452087},{"id":"https://openalex.org/keywords/gene-silencing","display_name":"gene silencing","score":0.7630000114440918},{"id":"https://openalex.org/keywords/cancer-cell-viability","display_name":"tumor cell viability","score":0.7730000019073486},{"id":"https://openalex.org/keywords/phosphorothioate-modification","display_name":"phosphorothioate modification","score":0.6909999847412109},{"id":"https://openalex.org/keywords/rna-delivery","display_name":"RNA delivery","score":0.3779999911785126},{"id":"https://openalex.org/keywords/rna-induced-silencing-complex","display_name":"RNA-induced silencing complex","score":0.6650000214576721},{"id":"https://openalex.org/keywords/dose-response-relationship","display_name":"dose-response relationship","score":0.36000001430511475}],"concepts":[{"id":"https://openalex.org/C166703698","wikidata":"https://www.wikidata.org/wiki/Q201993","display_name":"RNA interference","level":4,"score":0.7706054449081421},{"id":"https://openalex.org/C119056186","wikidata":"https://www.wikidata.org/wiki/Q1431332","display_name":"Gene silencing","level":3,"score":0.7204195261001587},{"id":"https://openalex.org/C2776577112","wikidata":"https://www.wikidata.org/wiki/Q500695","display_name":"Retinoblastoma","level":3,"score":0.713988721370697},{"id":"https://openalex.org/C67705224","wikidata":"https://www.wikidata.org/wiki/Q11053","display_name":"RNA","level":3,"score":0.5132057666778564},{"id":"https://openalex.org/C502942594","wikidata":"https://www.wikidata.org/wiki/Q3421914","display_name":"Cancer research","level":1,"score":0.5109788179397583},{"id":"https://openalex.org/C105580179","wikidata":"https://www.wikidata.org/wiki/Q188928","display_name":"Messenger RNA","level":3,"score":0.5011744499206543},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.4972217381000519},{"id":"https://openalex.org/C2781018059","wikidata":"https://www.wikidata.org/wiki/Q207609","display_name":"Oncogene","level":4,"score":0.46806493401527405},{"id":"https://openalex.org/C173396325","wikidata":"https://www.wikidata.org/wiki/Q1501275","display_name":"Gene knockdown","level":3,"score":0.4412330389022827},{"id":"https://openalex.org/C2776192174","wikidata":"https://www.wikidata.org/wiki/Q18028978","display_name":"Mdm2","level":3,"score":0.4367114305496216},{"id":"https://openalex.org/C22615655","wikidata":"https://www.wikidata.org/wiki/Q203221","display_name":"Small interfering RNA","level":4,"score":0.4218646287918091},{"id":"https://openalex.org/C153911025","wikidata":"https://www.wikidata.org/wiki/Q7202","display_name":"Molecular biology","level":1,"score":0.391055703163147},{"id":"https://openalex.org/C1491633281","wikidata":"https://www.wikidata.org/wiki/Q7868","display_name":"Cell","level":2,"score":0.3669864535331726},{"id":"https://openalex.org/C185592680","wikidata":"https://www.wikidata.org/wiki/Q2329","display_name":"Chemistry","level":0,"score":0.330846905708313},{"id":"https://openalex.org/C29537977","wikidata":"https://www.wikidata.org/wiki/Q188941","display_name":"Cell cycle","level":3,"score":0.3104720115661621},{"id":"https://openalex.org/C81885089","wikidata":"https://www.wikidata.org/wiki/Q189082","display_name":"Cell culture","level":2,"score":0.3070046901702881},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.259135365486145},{"id":"https://openalex.org/C54355233","wikidata":"https://www.wikidata.org/wiki/Q7162","display_name":"Genetics","level":1,"score":0.1613805890083313}],"mesh":[],"locations_count":1,"locations":[{"id":"mag:960773756","doi":null,"is_oa":false,"landing_page_url":"http://investors.rxipharma.com/file/4418218/Index?KeyFile=1500078826","pdf_url":null,"source":{"id":"https://openalex.org/S76304953","display_name":"Investigative Ophthalmology & Visual Science","issn_l":"0146-0404","issn":["0146-0404","1552-5783"],"is_oa":false,"is_in_doaj":true,"is_core":true,"listed_in":["anvur-scientific","cwts-core","doaj","dongbi-c","doyens","jufo-2","ki-jl-2","medline","norway-2","oarl","poland-140","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310321950","host_organization_name":"Cadmus Press","host_organization_lineage":["https://openalex.org/P4310321950"],"host_organization_lineage_names":["Cadmus Press"],"type":"journal"},"license":null,"license_id":null,"version":null,"is_accepted":false,"is_published":false,"raw_source_name":"Investigative Ophthalmology & Visual Science","raw_type":null}],"best_oa_location":null,"sustainable_development_goals":[{"score":0.8999999761581421,"display_name":"Good health and well-being","id":"https://metadata.un.org/sdg/3"}],"study_designs":[],"awards":[],"funders":[],"has_content":{"pdf":false,"grobid_xml":false},"content_urls":null,"referenced_works_count":0,"referenced_works":[],"related_works":["https://openalex.org/W2359222910","https://openalex.org/W1001312357","https://openalex.org/W2416672790","https://openalex.org/W2022929716","https://openalex.org/W7100550535","https://openalex.org/W1510019806","https://openalex.org/W2624514106","https://openalex.org/W3025797153","https://openalex.org/W3176185887","https://openalex.org/W2340740689"],"abstract_inverted_index":{"Figure":[0,100,142,153,286,845],"5.":[1],"MDM2-targeting":[2,42,76,94,510,533,574,637],"sd-rxRNAs":[3,43,77,166,498,511,534,575,593,638,657,773,792],"Significantly":[4],"Reduce":[5],"MDM2":[6,16,48,233,260,448,501,515,545,554,577,583,591,611,626,655],"mRNA":[7,49,136,234,261,516,546,578,612,644,810,825],"Levels":[8],"in":[9,53,63,79,125,249,292,294,308,347,369,427,475,481,491,502,541,617,641,793,814,850,852,898,908,915],"vitro":[10,293,794],"Through":[11],"Day":[12,623,634],"6":[13,186],"Conclusions":[14],"\u2022":[15,41,70,86],"is":[17,32,110,342,354,433,454,466,913],"known":[18],"to":[19,34,133,389,392,434,439,468,660,670,755,764,776,820],"be":[20,390,402],"a":[21,111,173,181,266,343,409,450,455,492,550,603,618,779,815,830,839,843],"crucial":[22,456],"component":[23,457],"of":[24,37,59,72,93,103,117,148,163,172,225,232,321,333,350,366,374,379,383,412,421,458,471,484,651,662,681,686,695,848,877],"the":[25,38,90,128,164,348,364,370,436,459,472,692,899,909,916],"cone":[26,380,460],"circuitry":[27,373,461],"underlying":[28,462],"retinoblastoma":[29,97,393,443,463,476,489,504,608,671,698],"development":[30,464],"and":[31,50,138,150,180,197,208,251,276,302,396,423,425,465,495,517,528,555,599,627,645,683,801,855],"critical":[33,391],"sustained":[35],"expression":[36,365,470,527,598],"MYCN":[39,65,473,525,556,596,628,663],"oncogene.":[40],"were":[44,217,238,242,263,278,539,559,614,631,639,648,774,787,812,827,865],"identified":[45,496,535],"that":[46,345,378,399,418,499],"reduce":[47],"protein":[51,66,518,526,557,584,597,629,646,664],"levels":[52,67,262,558,579,585,613,630,665,811,826],"vitro.":[54,642],"Importantly,":[55,588],"MDM2targeting":[56],"sd-rxRNA":[57,95,144,156,190,437,486,696,708,730,740,834,849,879,891,903,912],"treatment":[58,589],"RB176":[60,73,250,543,769],"cells":[61,74,122,213,490,653,784,896,907],"resulted":[62,78],"reduced":[64,80,209,576,616,632,813],"over":[68,84,586],"time.":[69,85,587],"Treatment":[71,650],"with":[75,127,195,219,229,590,654,677,789,868,894,905],"tumor":[81,895,906],"cell":[82,253,394,529,600,609,672],"viability":[83],"These":[87,674],"findings":[88],"support":[89,691],"potential":[91,604,693],"use":[92,435,694],"for":[96,160,244,280,330,336,512,570,697],"therapy.":[98,699],"Background":[99],"1:":[101],"Mechanism":[102],"Cellular":[104],"Gene":[105],"Silencing":[106,291],"RNA":[107,120],"interference":[108],"(RNAi)":[109],"naturally":[112],"occurring":[113],"cellular":[114,206,429],"process.":[115],"Introduction":[116],"double":[118],"stranded":[119],"into":[121],"can":[123,668],"result":[124],"association":[126],"RNA-induced":[129],"silencing":[130,307,685],"complex":[131],"(RISC)":[132],"target":[134,140,451,777],"complementary":[135],"sequences":[137],"degrade":[139],"genes.":[141],"2:":[143],"Incorporates":[145],"Advanced":[146],"Features":[147],"RNAi":[149,169,322,334,415,422],"Antisense":[151],"Technologies":[152],"4:":[154],"MDM2-Targeting":[155],"Selection":[157],"E-mail":[158],"mbyrne@rxipharma.com":[159],"PDF":[161],"version":[162],"poster":[165],"are":[167,192],"asymmetric":[168],"compounds":[170,191,228,241,274,416,441],"comprised":[171],"small":[174],"duplex":[175],"region":[176],"(<15":[177],"base":[178],"pairs)":[179],"single-stranded":[182],"phosphorothioate":[183],"tail":[184],"(\u2265":[185],"nucleotides).":[187],"In":[188],"addition,":[189],"chemically":[193],"modified":[194],"stabilizing":[196],"hydrophobic":[198],"modifications":[199],"(e.g.,":[200],"sterol),":[201],"which":[202,666],"confer":[203],"stability,":[204],"efficient":[205],"uptake":[207,305,483],"inflammatory":[210],"response.":[211],"RB177":[212,252,771],"(50,000":[214],"per":[215,785],"well)":[216],"treated":[218,788],"0.05,":[220,798],"0.1":[221,714],"or":[222,759],"0.5":[223,236],"uM":[224,237,711,713,715,717,719,721],"compound.":[226],"Seven":[227],"~40-50%":[230],"reduction":[231,661],"at":[235,752,795],"identified.":[239],"Four":[240],"chosen":[243,279],"6-point":[245],"dose":[246,619,816],"response":[247],"studies":[248,479,538],"lines.":[254],"At":[255,804],"48":[256,805],"hours":[257,806,859,887],"post":[258,807,873,888],"administration":[259,808],"quantified":[264,828],"by":[265,356,363,487,561,568,689,829,883],"branched":[267,551,831],"DNA":[268,552,832],"assay.":[269,553,833],"From":[270],"these":[271,384],"screens":[272],"two":[273,509],"(25799":[275],"25805)":[277],"further":[281],"analysis.":[282],"NTC=":[283],"non-targeting":[284,821],"sd-rxRNA.":[285,824],"3:":[287,846],"sd-rxRNA:":[288],"Dose":[289],"Dependent":[290],"Retinoblastoma":[295,341],"Cell":[296,564],"Lines":[297],"Single":[298],"compound":[299],"incorporates":[300],"activity":[301],"delivery":[303],"Robust":[304],"&":[306,323],"multiple":[309],"preclinical":[310],"models":[311],"Structural":[312],"diversity":[313],"=":[314],"novel":[315],"intellectual":[316],"property":[317],"Combining":[318],"many":[319,327],"positives":[320],"antisense,":[324],"while":[325],"avoiding":[326],"negatives":[328],"Provides":[329],"broad":[331],"pipeline":[332],"drugs":[335],"unmet":[337],"medical":[338],"needs":[339],"Purpose:":[340],"cancer":[344],"originates":[346],"retina":[349],"young":[351],"children.":[352],"It":[353],"driven":[355],"inactivating":[357],"RB1":[358],"mutations,":[359],"as":[360,362,449,520,522,580,582],"well":[361,521,581,786],"genes":[367,385,688],"involved":[368],"\u201cnormal\u201d":[371],"signaling":[372],"retinal":[375,687],"cells,":[376],"particularly":[377],"precursors.":[381],"Some":[382],"have":[386],"been":[387],"found":[388],"growth":[395],"survival,":[397],"suggesting":[398,602],"they":[400],"may":[401],"effective":[403],"therapeutic":[404,444,605],"targets.":[405,445],"RXi":[406],"has":[407],"developed":[408],"new":[410],"class":[411],"stable,":[413],"self-delivering":[414],"(sd-rxRNA\u00ae)":[417],"incorporate":[419],"features":[420],"antisense":[424],"results":[426],"spontaneous":[428],"uptake.":[430],"Our":[431],"goal":[432],"platform":[438],"develop":[440],"against":[442],"We":[446],"selected":[447],"because":[452],"it":[453],"required":[467],"sustain":[469],"oncogene":[474],"cells.":[477,505,544,870],"Initial":[478],"demonstrated":[480],"vivo":[482,851],"control":[485,822],"human":[488],"mouse":[493],"model":[494],"candidate":[497],"down-regulated":[500],"cultured":[503,542,652],"Here,":[506],"we":[507],"evaluated":[508,540,548,567,640],"effects":[513],"on":[514,524,607],"expression,":[519],"impact":[523,606],"proliferation.":[530],"Methods:":[531],"Two":[532],"from":[536],"screening":[537],"was":[547,566,881],"using":[549],"determined":[560],"Western":[562],"blot.":[563],"proliferation":[565],"assaying":[569],"ATP":[571],"production.":[572],"Results:":[573],"targeting":[592,656,791],"also":[594,658],"decreased":[595],"proliferation,":[601],"viability.":[610],"significantly":[615],"dependent":[620,817],"manner":[621,818],"through":[622,633],"6.":[624],"Moreover,":[625],"7.":[635],"Conclusions:":[636],"Target-specific":[643],"reductions":[647],"observed.":[649],"led":[659],"importantly":[667],"lead":[669],"death.":[673],"findings,":[675],"along":[676],"our":[678],"previous":[679],"report":[680],"specific":[682],"extended":[684],"sd-rxRNA,":[690],"0":[700,722,732,918],"0.2":[701,723,733],"0.4":[702,724,734],"0.6":[703,725,735],"0.8":[704,726,736],"1":[705,710,727,737,802],"1.2":[706,728,738],"PPIB":[707,729,739,790,809],"NTC":[709,731,741],"0.3":[712],"0.05":[716],"0.025":[718],"0.01":[720],"PP":[742],"IB":[743],"m":[744,760],"RN":[745],"A":[746],"Le":[747],"ve":[748],"ls":[749],"R":[750],"el":[751],"iv":[753],"e":[754],"N":[756],"TC":[757],"(N":[758],"al":[761],"ize":[762],"d":[763],"G":[765],"AP":[766],"DH":[767],")":[768],"Y79":[770,869],"Model":[772],"designed":[775],"PPIB,":[778],"ubiquitously":[780],"expressed":[781],"gene.":[782],"50,000":[783],"0.01,":[796],"0.025,":[797],"0.1,":[799],"0.3,":[800],"uM.":[803],"relative":[819],"(NTC)":[823],"sd-rxRNA/Cone":[835],"arrestin":[836,838],"Cone":[837],"c":[840,842],"b":[841,844],"Uptake":[847],"Mouse":[853,863],"Retina":[854],"Tumor":[856],"Cells":[857],"24":[858],"Post":[860],"Intravitreal":[861],"Injection":[862],"eyes":[864],"seeded":[866],"subretinally":[867],"Three":[871],"weeks":[872],"seeding,":[874],"10":[875],"ug":[876],"fluorescent":[878],"(red)":[880,892],"administered":[882],"intravitreal":[884],"injection.":[885],"Twenty-four":[886],"injection":[889],"a)":[890],"co-localized":[893],"(green)":[897],"subretinal":[900],"space;":[901],"b)":[902],"localized":[904],"vitreous;":[910],"c)":[911],"visible":[914],"retina.":[917],"20":[919],"40":[920],"60":[921],"80":[922],"100":[923],"120":[924],"14":[925]},"counts_by_year":[],"updated_date":"2026-10-22T18:32:59.640997","created_date":"2016-06-24T00:00:00"}