Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing

AUTOR(ES)

Sazani, Peter

FONTE

American Society for Clinical Investigation

RESUMO

An estimated 60% of all human genes undergo alternative splicing, a highly regulated process that produces 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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