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A deep learning approach to identify gene targets of a therapeutic for human splicing disorders

Pre-mRNA splicing is a key controller of human gene expression. Disturbances in splicing due to mutation lead to dysregulated protein expression and contribute to a substantial fraction of human disease. Several classes of splicing modulator compounds (SMCs) have been recently identified and establi...

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Detalles Bibliográficos
Autores principales: Gao, Dadi, Morini, Elisabetta, Salani, Monica, Krauson, Aram J., Chekuri, Anil, Sharma, Neeraj, Ragavendran, Ashok, Erdin, Serkan, Logan, Emily M., Li, Wencheng, Dakka, Amal, Narasimhan, Jana, Zhao, Xin, Naryshkin, Nikolai, Trotta, Christopher R., Effenberger, Kerstin A., Woll, Matthew G., Gabbeta, Vijayalakshmi, Karp, Gary, Yu, Yong, Johnson, Graham, Paquette, William D., Cutting, Garry R., Talkowski, Michael E., Slaugenhaupt, Susan A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8185002/
https://www.ncbi.nlm.nih.gov/pubmed/34099697
http://dx.doi.org/10.1038/s41467-021-23663-2