ProjectGeko Invest – RNA-encoded and RNA-based site specific nucleases for in vivo gene correction of the hCFTR delTT394…
Basic data
Acronym:
Geko Invest
Title:
RNA-encoded and RNA-based site specific nucleases for in vivo gene correction of the hCFTR delTT394 "nordic" mutation
Duration:
01/07/2021 to 30/06/2022
Abstract / short description:
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.
Involved staff
Managers
Faculty of Medicine
University of Tübingen
University of Tübingen
Local organizational units
University Children’s Hospital - Department of Paediatrics
Hospitals and clinical institutes
Faculty of Medicine
Faculty of Medicine
Funders
Pfaffstätten, Austria