Repair of thalassemic human β-globin mRNA in mammalian cells by antisense oligonucleotides

In one form of β-thalassemia, a genetic blood disorder, a mutation in intron 2 of the β-globin gene (IVS2-654) causes aberrant splicing of β-globin pre-mRNA and, consequently, β-globin deficiency. Treatment of mammalian cells stably expressing the IVS2-654 human β-globin gene with antisense oligonuc...

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Dades bibliogràfiques

Publicat a:	Proc Natl Acad Sci U S A
Autors principals:	Sierakowska, Halina, Sambade, Maria J., Agrawal, Sudhir, Kole, Ryszard
Format:	Artigo
Idioma:	Inglês
Publicat:	National Academy of Sciences 1996
Matèries:	Biological Sciences
Accés en línia:	https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC24007/ https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/8917506/ https://ncbi.nlm.nih.govhttps://doi.org/10.1073/pnas.93.23.12840
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