ProjektGeko Invest – RNA-encoded and RNA-based site specific nucleases for in vivo gene correction of the hCFTR delTT394…
Grunddaten
Akronym:
Geko Invest
Titel:
RNA-encoded and RNA-based site specific nucleases for in vivo gene correction of the hCFTR delTT394 "nordic" mutation
Laufzeit:
01.07.2021 bis 30.06.2022
Abstract / Kurz- beschreibung:
Cystic fibrosis (CF) is a hereditary and life-threatening disorder with persistent lung infections. This disease is caused by mutations in the cystic fibrosis transmembrane and conductance regulator (CFTR) gene and the resulting of dysfunctional CFTR protein.
Based on the extensive experience of our team in utilizing gene therapy in vivo and gene editing ex vivo and in vivo, the achievment is to acquire a design and construction of the gene correction technology to target the CFTR gene mutation (delTT394) in the humanized CF mouse model. Furthermore with focusing on CRISPR/Cas9 design obtaining a permanent and sufficient CFTR expression in the lungs in vivo.
Based on the extensive experience of our team in utilizing gene therapy in vivo and gene editing ex vivo and in vivo, the achievment is to acquire a design and construction of the gene correction technology to target the CFTR gene mutation (delTT394) in the humanized CF mouse model. Furthermore with focusing on CRISPR/Cas9 design obtaining a permanent and sufficient CFTR expression in the lungs in vivo.
Beteiligte Mitarbeiter/innen
Leiter/innen
Medizinische Fakultät
Universität Tübingen
Universität Tübingen
Lokale Einrichtungen
Universitätsklinik für Kinder- und Jugendmedizin (Department)
Kliniken und klinische Institute
Medizinische Fakultät
Medizinische Fakultät
Geldgeber
Pfaffstätten, Österreich