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Efficient and specific repair of sickle β-globin RNA by trans-splicing ribozymes

Previously we demonstrated that a group I ribozyme can perform trans-splicing to repair sickle β-globin transcripts upon transfection of in vitro transcribed ribozyme into mammalian cells. Here, we sought to develop expression cassettes that would yield high levels of active ribozyme after gene tran...

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Detaylı Bibliyografya
Asıl Yazarlar: BYUN, JONGHOE, LAN, NING, LONG, MEREDITH, SULLENGER, BRUCE A.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: Copyright 2003 by RNA Society 2003
Konular:
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC1370489/
https://ncbi.nlm.nih.gov/pubmed/13130139
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1261/rna.5450203
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