{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T05:19:29Z","timestamp":1790831969135,"version":"4.1.0"},"reference-count":121,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2021,2,23]],"date-time":"2021-02-23T00:00:00Z","timestamp":1614038400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100004281","name":"Narodowe Centrum Nauki","doi-asserted-by":"publisher","award":["UMO\u20102016\/21\/B\/NZ1\/00232"],"award-info":[{"award-number":["UMO\u20102016\/21\/B\/NZ1\/00232"]}],"id":[{"id":"10.13039\/501100004281","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004281","name":"Narodowe Centrum Nauki","doi-asserted-by":"publisher","award":["UMO\u20102018\/30\/E\/NZ2\/00295"],"award-info":[{"award-number":["UMO\u20102018\/30\/E\/NZ2\/00295"]}],"id":[{"id":"10.13039\/501100004281","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["The Journal of Gene Medicine"],"published-print":{"date-parts":[[2021,4]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Most U\u2010rich small nuclear ribonucleoproteins (snRNPs) are complexes that mediate the splicing of pre\u2010mRNAs. U7 snRNP is an exception in that it is not involved in splicing but is a key factor in the unique 3\u2032 end processing of replication\u2010dependent histone mRNAs. However, by introducing controlled changes in the U7 snRNA histone binding sequence and in the Sm motif, it can be used as an effective tool for gene therapy. The modified U7 snRNP (U7 Sm OPT) is thus not involved in the processing of replication\u2010dependent histone pre\u2010mRNA but targets splicing by inducing efficient skipping or inclusion of selected exons. U7 Sm OPT is of therapeutic importance in diseases that are an outcome of splicing defects, such as myotonic dystrophy, Duchenne muscular dystrophy, amyotrophic lateral sclerosis, \u03b2\u2010thalassemia, HIV\u20101 infection and spinal muscular atrophy. The benefits of using U7 Sm OPT for gene therapy are its compact size, ability to accumulate in the nucleus without causing any toxic effects in the cells, and no immunoreactivity. The risk of transgene misregulation by using U7 Sm OPT is also low because it is involved in correcting the expression of an endogenous gene controlled by its own regulatory elements. Altogether, using U7 Sm OPT as a tool in gene therapy can ensure lifelong treatment, whereas an oligonucleotide or other drug\/compound would require repeated administration. It would thus be strategic to harness these unique properties of U7 snRNP and deploy it as a tool in gene therapy.<\/jats:p>","DOI":"10.1002\/jgm.3321","type":"journal-article","created":{"date-parts":[[2021,2,16]],"date-time":"2021-02-16T04:00:13Z","timestamp":1613448013000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":54,"title":["U7 snRNA: A tool for gene therapy"],"prefix":"10.1002","volume":"23","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-5155-4079","authenticated-orcid":false,"given":"Ankur","family":"Gadgil","sequence":"first","affiliation":[{"name":"Department of Gene Expression, Institute of Molecular Biology and Biotechnology, Faculty of Biology Adam Mickiewicz University  Poznan Poland"},{"name":"Center for Advanced Technology Adam Mickiewicz University  Poznan Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3712-3528","authenticated-orcid":false,"given":"Katarzyna Dorota","family":"Raczy\u0144ska","sequence":"additional","affiliation":[{"name":"Department of Gene Expression, Institute of Molecular Biology and Biotechnology, Faculty of Biology Adam Mickiewicz University  Poznan Poland"},{"name":"Center for Advanced Technology Adam Mickiewicz University  Poznan Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2021,2,23]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/0022-2836(68)90387-2"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.1038\/283220a0"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.3892\/br.2014.407"},{"key":"e_1_2_9_5_1","doi-asserted-by":"publisher","DOI":"10.1038\/271501a0"},{"key":"e_1_2_9_6_1","doi-asserted-by":"publisher","DOI":"10.1126\/science.282.5396.2012"},{"key":"e_1_2_9_7_1","doi-asserted-by":"publisher","DOI":"10.1038\/nature03001"},{"key":"e_1_2_9_8_1","doi-asserted-by":"publisher","DOI":"10.1261\/rna.876308"},{"key":"e_1_2_9_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.molcel.2006.05.018"},{"key":"e_1_2_9_10_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.75.1.285"},{"key":"e_1_2_9_11_1","doi-asserted-by":"publisher","DOI":"10.1093\/nar\/gkx1239"},{"key":"e_1_2_9_12_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.90.18.8673"},{"key":"e_1_2_9_13_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ceb.2005.04.010"},{"key":"e_1_2_9_14_1","doi-asserted-by":"publisher","DOI":"10.1261\/rna.782308"},{"key":"e_1_2_9_15_1","doi-asserted-by":"publisher","DOI":"10.1093\/bfgp\/ell020"},{"key":"e_1_2_9_16_1","doi-asserted-by":"publisher","DOI":"10.1128\/MCB.11.5.2416"},{"key":"e_1_2_9_17_1","doi-asserted-by":"publisher","DOI":"10.1006\/scdb.1997.0182"},{"key":"e_1_2_9_18_1","doi-asserted-by":"publisher","DOI":"10.1101\/gad.10.23.3028"},{"key":"e_1_2_9_19_1","doi-asserted-by":"publisher","DOI":"10.1126\/science.2825355"},{"key":"e_1_2_9_20_1","doi-asserted-by":"publisher","DOI":"10.1101\/gad.5.9.1709"},{"key":"e_1_2_9_21_1","doi-asserted-by":"publisher","DOI":"10.1038\/323777a0"},{"key":"e_1_2_9_22_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.82.13.4389"},{"key":"e_1_2_9_23_1","doi-asserted-by":"publisher","DOI":"10.1038\/nrg2438"},{"key":"e_1_2_9_24_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.cell.2005.08.002"},{"key":"e_1_2_9_25_1","doi-asserted-by":"publisher","DOI":"10.1128\/MCB.00653-12"},{"key":"e_1_2_9_26_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.tig.2017.07.014"},{"key":"e_1_2_9_27_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ceb.2016.01.015"},{"key":"e_1_2_9_28_1","doi-asserted-by":"publisher","DOI":"10.1091\/mbc.e10-06-0543"},{"key":"e_1_2_9_29_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00018-004-4190-0"},{"key":"e_1_2_9_30_1","doi-asserted-by":"publisher","DOI":"10.1093\/emboj\/20.19.5470"},{"key":"e_1_2_9_31_1","doi-asserted-by":"publisher","DOI":"10.1101\/gad.274403"},{"key":"e_1_2_9_32_1","doi-asserted-by":"publisher","DOI":"10.1128\/MCB.8.3.1076"},{"key":"e_1_2_9_33_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.1533509100"},{"key":"e_1_2_9_34_1","doi-asserted-by":"publisher","DOI":"10.1074\/jbc.M505077200"},{"key":"e_1_2_9_35_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.celrep.2013.11.012"},{"key":"e_1_2_9_36_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.gene.2007.04.021"},{"key":"e_1_2_9_37_1","doi-asserted-by":"publisher","DOI":"10.1002\/j.1460-2075.1993.tb05764.x"},{"key":"e_1_2_9_38_1","doi-asserted-by":"publisher","DOI":"10.1093\/nar\/23.16.3141"},{"key":"e_1_2_9_39_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbrc.2007.04.172"},{"key":"e_1_2_9_40_1","doi-asserted-by":"publisher","DOI":"10.4161\/rna.8.3.15388"},{"key":"e_1_2_9_41_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.febslet.2010.09.039"},{"key":"e_1_2_9_42_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.gde.2011.04.001"},{"key":"e_1_2_9_43_1","doi-asserted-by":"publisher","DOI":"10.1017\/S1355838202024056"},{"key":"e_1_2_9_44_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/8.13.2415"},{"key":"e_1_2_9_45_1","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2009.113"},{"key":"e_1_2_9_46_1","doi-asserted-by":"publisher","DOI":"10.1038\/sj.mt.6300200"},{"key":"e_1_2_9_47_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddn382"},{"key":"e_1_2_9_48_1","doi-asserted-by":"publisher","DOI":"10.1089\/104454902762053855"},{"key":"e_1_2_9_49_1","doi-asserted-by":"publisher","DOI":"10.1038\/ng0393-224"},{"key":"e_1_2_9_50_1","first-page":"1502","volume-title":"Fields virology","author":"Fields BN","year":"2013"},{"key":"e_1_2_9_51_1","doi-asserted-by":"publisher","DOI":"10.2174\/1566523214666140302193709"},{"key":"e_1_2_9_52_1","doi-asserted-by":"publisher","DOI":"10.1038\/gt.2012.6"},{"key":"e_1_2_9_53_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10038-007-0192-8"},{"key":"e_1_2_9_54_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nrl.2015.12.009"},{"key":"e_1_2_9_55_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.0633863100"},{"key":"e_1_2_9_56_1","doi-asserted-by":"publisher","DOI":"10.1038\/nsb887"},{"key":"e_1_2_9_57_1","doi-asserted-by":"publisher","DOI":"10.1016\/0092-8674(92)90154-5"},{"key":"e_1_2_9_58_1","doi-asserted-by":"publisher","DOI":"10.1038\/nsmb.1958"},{"key":"e_1_2_9_59_1","doi-asserted-by":"publisher","DOI":"10.1007\/978-1-61737-982-6_11"},{"key":"e_1_2_9_60_1","doi-asserted-by":"publisher","DOI":"10.1126\/science.1104297"},{"key":"e_1_2_9_61_1","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2013.121"},{"key":"e_1_2_9_62_1","doi-asserted-by":"publisher","DOI":"10.1038\/nm0302-253"},{"key":"e_1_2_9_63_1","doi-asserted-by":"publisher","DOI":"10.1002\/humu.20367"},{"key":"e_1_2_9_64_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtn.2017.02.004"},{"key":"e_1_2_9_65_1","doi-asserted-by":"publisher","DOI":"10.1038\/nm.3628"},{"key":"e_1_2_9_66_1","doi-asserted-by":"publisher","DOI":"10.1007\/s000180300047"},{"key":"e_1_2_9_67_1","doi-asserted-by":"publisher","DOI":"10.1002\/jgm.295"},{"key":"e_1_2_9_68_1","doi-asserted-by":"publisher","DOI":"10.1006\/mthe.2000.0219"},{"key":"e_1_2_9_69_1","doi-asserted-by":"publisher","DOI":"10.1007\/978-1-61779-767-5_17"},{"key":"e_1_2_9_70_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2004.05.031"},{"key":"e_1_2_9_71_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/dds082"},{"key":"e_1_2_9_72_1","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2012.181"},{"key":"e_1_2_9_73_1","doi-asserted-by":"publisher","DOI":"10.1016\/0888-7543(92)90210-J"},{"key":"e_1_2_9_74_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40265-016-0657-1"},{"key":"e_1_2_9_75_1","first-page":"272","article-title":"Therapeutic approach with Ataluren in Duchenne symptomatic carriers with nonsense mutations in dystrophin gene. Results of a 9\u2010month follow\u2010up in a case report","volume":"37","author":"D'Ambrosio P","year":"2018","journal-title":"Acta Myol"},{"key":"e_1_2_9_76_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41591-019-0594-0"},{"key":"e_1_2_9_77_1","doi-asserted-by":"publisher","DOI":"10.3390\/ijms19051345"},{"key":"e_1_2_9_78_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0022-510X(99)00216-6"},{"key":"e_1_2_9_79_1","doi-asserted-by":"publisher","DOI":"10.1038\/362059a0"},{"key":"e_1_2_9_80_1","doi-asserted-by":"publisher","DOI":"10.1136\/jnnp-2015-310545"},{"key":"e_1_2_9_81_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2017.05.017"},{"key":"e_1_2_9_82_1","doi-asserted-by":"publisher","DOI":"10.1093\/nar\/gku184"},{"key":"e_1_2_9_83_1","doi-asserted-by":"publisher","DOI":"10.1101\/cshperspect.a011734"},{"key":"e_1_2_9_84_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbrc.2018.03.102"},{"key":"e_1_2_9_85_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.81.9.2821"},{"key":"e_1_2_9_86_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41598-019-43964-3"},{"key":"e_1_2_9_87_1","doi-asserted-by":"publisher","DOI":"10.1002\/sctm.16-0121"},{"key":"e_1_2_9_88_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.93.23.12840"},{"key":"e_1_2_9_89_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.95.9.4929"},{"key":"e_1_2_9_90_1","doi-asserted-by":"publisher","DOI":"10.1007\/BF00291231"},{"key":"e_1_2_9_91_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2141.1994.tb05071.x"},{"key":"e_1_2_9_92_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2002-06-1869"},{"key":"e_1_2_9_93_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.mib.2010.06.002"},{"key":"e_1_2_9_94_1","doi-asserted-by":"publisher","DOI":"10.1002\/jgm.1027"},{"key":"e_1_2_9_95_1","doi-asserted-by":"publisher","DOI":"10.1002\/jgm.1238"},{"key":"e_1_2_9_96_1","doi-asserted-by":"publisher","DOI":"10.1093\/nar\/gkh715"},{"key":"e_1_2_9_97_1","doi-asserted-by":"publisher","DOI":"10.1038\/372363a0"},{"key":"e_1_2_9_98_1","doi-asserted-by":"publisher","DOI":"10.1006\/geno.2000.6295"},{"key":"e_1_2_9_99_1","doi-asserted-by":"publisher","DOI":"10.1002\/(SICI)1098-1004(200003)15:3<228::AID-HUMU3>3.0.CO;2-9"},{"key":"e_1_2_9_100_1","doi-asserted-by":"publisher","DOI":"10.1038\/344540a0"},{"key":"e_1_2_9_101_1","doi-asserted-by":"publisher","DOI":"10.1016\/0092-8674(95)90460-3"},{"key":"e_1_2_9_102_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.96.11.6307"},{"key":"e_1_2_9_103_1","doi-asserted-by":"publisher","DOI":"10.1016\/0092-8674(95)90442-5"},{"key":"e_1_2_9_104_1","doi-asserted-by":"publisher","DOI":"10.4161\/rna.7.4.12206"},{"key":"e_1_2_9_105_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2005.08.022"},{"key":"e_1_2_9_106_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2010.06.010"},{"key":"e_1_2_9_107_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/9.3.333"},{"key":"e_1_2_9_108_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0008204"},{"key":"e_1_2_9_109_1","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2016.152"},{"key":"e_1_2_9_110_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40265-017-0711-7"},{"key":"e_1_2_9_111_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.clinthera.2013.11.006"},{"key":"e_1_2_9_112_1","doi-asserted-by":"publisher","DOI":"10.1146\/annurev-neuro-070918-050501"},{"key":"e_1_2_9_113_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1710504"},{"key":"e_1_2_9_114_1","first-page":"113","article-title":"Zolgensma \u2010 one\u2010time gene therapy for spinal muscular atrophy","volume":"61","year":"2019","journal-title":"Med Lett Drugs Ther"},{"key":"e_1_2_9_115_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1706198"},{"key":"e_1_2_9_116_1","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2020.145"},{"key":"e_1_2_9_117_1","doi-asserted-by":"publisher","DOI":"10.1038\/mtna.2012.24"},{"key":"e_1_2_9_118_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jconrel.2004.08.027"},{"key":"e_1_2_9_119_1","doi-asserted-by":"publisher","DOI":"10.1093\/nar\/gkn342"},{"key":"e_1_2_9_120_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtn.2020.01.018"},{"key":"e_1_2_9_121_1","doi-asserted-by":"publisher","DOI":"10.3390\/genes8020051"},{"key":"e_1_2_9_122_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41573-020-0075-7"}],"container-title":["The Journal of Gene Medicine"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/jgm.3321","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/jgm.3321","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/jgm.3321","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,9,1]],"date-time":"2023-09-01T21:24:20Z","timestamp":1693603460000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/jgm.3321"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,2,23]]},"references-count":121,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2021,4]]}},"alternative-id":["10.1002\/jgm.3321"],"URL":"https:\/\/doi.org\/10.1002\/jgm.3321","archive":["Portico"],"relation":{},"ISSN":["1099-498X","1521-2254"],"issn-type":[{"value":"1099-498X","type":"print"},{"value":"1521-2254","type":"electronic"}],"subject":[],"published":{"date-parts":[[2021,2,23]]},"assertion":[{"value":"2020-06-09","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2021-02-09","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2021-02-23","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}],"article-number":"e3321"}}