CCLS Webinar: Computational approaches for the development of gene therapies
- Tuesday 18 May 2021
Computational approaches for the development of gene therapies
André Leier, University of Alabama, USA
I will present recent work by us and our collaborators to develop gene therapies for the treatment of genetic diseases, including Neurofibromatosis Type I (NF1) and Cystic Fibrosis, caused by mutations in the genes neurofibromin (NF1) and CFTR (cystic fibrosis transmembrane conductance regulator), respectively. Specifically, I will describe bioinformatics efforts that led to the identification of promising target exons for therapeutic exon skipping in NF1 as well as computational and experimental approaches for optimizing trans-splicing ribozymes, i.e. catalytic RNAs, that repair mutated NF1 and CFTR (pre-)mRNA.
For a link to the meeting, please contact Chris Flinterman.