Journal
JOURNAL OF CLINICAL INVESTIGATION
Volume 112, Issue 4, Pages 481-486Publisher
AMER SOC CLINICAL INVESTIGATION INC
DOI: 10.1172/JCI200319547
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Funding
- NHLBI NIH HHS [HL-51940] Funding Source: Medline
- NIGMS NIH HHS [P01-GM59299, P01 GM059299] Funding Source: Medline
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An estimated 60% of all human genes undergo alternative splicing, a highly regulated process that pro, duces splice variants with different functions. Such variants have been linked to a variety of cancers, and genetic diseases such as thalassemia and cystic fibrosis. This Perspective describes a promising approach to RNA repair based on the use of antisense oligonucleotides to modulate alternative splicing and engender the production of therapeutic gene products.
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