Therapeutic potential of splice-switching oligonucleotides

• Main Authors: John Bauman, Natee Jearawiriyapaisarn, Ryszard Kole
• Other Authors: The University of North Carolina at Chapel Hill
• Format: Review
• Published: 2018
• Subjects: Biochemistry, Genetics and Molecular Biology
• Online Access: https://repository.li.mahidol.ac.th/handle/123456789/27275
• Institution: Mahidol University

Alternative splicing enables a single pre-messenger RNA transcript to yield multiple protein isoforms, making it a major contributor to the diversity of the proteome. While this process is essential for normal development, aberrations in alternative splicing are the cause of a multitude of human dis...