Therapeutic potential of splice-switching oligonucleotides
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...
Saved in:
Main Authors: | John Bauman, Natee Jearawiriyapaisarn, Ryszard Kole |
---|---|
Other Authors: | The University of North Carolina at Chapel Hill |
Format: | Review |
Published: |
2018
|
Subjects: | |
Online Access: | https://repository.li.mahidol.ac.th/handle/123456789/27275 |
Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
Institution: | Mahidol University |
Similar Items
-
Engineered U7 Small Nuclear RNA Restores Correct β-Globin Pre-mRNA Splicing in Mouse β<sup>iVS2-654</sup>-Thalassemic Erythroid Progenitor Cells
by: Annette D'Arqom, et al.
Published: (2022) -
Engineered U7 snRNA mediates sustained splicing correction in erythroid cells from β-thalassemia/HbE patients
by: Sarinthip Preedagasamzin, et al.
Published: (2019) -
Peptide-morpholino conjugate: A promising therapeutic for duchenne muscular dystrophy
by: Hong M. Moulton, et al.
Published: (2018) -
Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers
by: Natee Jearawiriyapaisarn, et al.
Published: (2018) -
Repair of a splicing defect in erythroid cells from patients with β-thalassemia/HbE disorder
by: Thipparat Suwanmanee, et al.
Published: (2018)