Universiteit Leiden

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Lecture

CCLS Webinar: Computational approaches for the development of gene therapies

Date
Tuesday 18 May 2021
Time
Location
Online

Computational approaches for the development of gene therapies

André Leier, University of Alabama, USA

Abstract:
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.

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