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Both\n products persisted for up to 72 hr posttreatment. The oligonucleotides\n modified splicing by a true antisense mechanism without overt\n unspecific effects on cell growth and splicing of other pre-mRNAs. This\n novel approach in which antisense oligonucleotides are used to restore\n rather than to down-regulate the activity of the target gene is\n applicable to other splicing mutants and is of potential clinical\n interest.<\/jats:p>","DOI":"10.1073\/pnas.93.23.12840","type":"journal-article","created":{"date-parts":[[2002,7,26]],"date-time":"2002-07-26T10:31:44Z","timestamp":1027679504000},"page":"12840-12844","update-policy":"https:\/\/doi.org\/10.1073\/pnas.cm10313","source":"Crossref","is-referenced-by-count":200,"title":["Repair of thalassemic human \u03b2-globin mRNA in mammalian cells by antisense\u2009oligonucleotides"],"prefix":"10.1073","volume":"93","author":[{"given":"Halina","family":"Sierakowska","sequence":"first","affiliation":[{"name":"Lineberger Comprehensive Cancer Center and Department of\r Pharmacology, University of North Carolina, Chapel Hill, NC 27599; and\r Hybridon, Inc., Worcester, MA 01605"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maria\u2009J.","family":"Sambade","sequence":"additional","affiliation":[{"name":"Lineberger Comprehensive Cancer Center and Department of\r Pharmacology, University of North Carolina, Chapel Hill, NC 27599; and\r Hybridon, Inc., Worcester, MA 01605"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sudhir","family":"Agrawal","sequence":"additional","affiliation":[{"name":"Lineberger Comprehensive Cancer Center and Department of\r Pharmacology, University of North Carolina, Chapel Hill, NC 27599; and\r Hybridon, Inc., Worcester, MA 01605"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ryszard","family":"Kole","sequence":"additional","affiliation":[{"name":"Lineberger Comprehensive Cancer Center and Department of\r Pharmacology, University of North Carolina, Chapel Hill, NC 27599; and\r Hybridon, Inc., Worcester, MA 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