Project mRNA lung cure – Demonstration of a Cas9 mRNA-mediated rescue of SP-B def. mice after i.t. delivery; detailed…

Basic data

Acronym:
mRNA lung cure
Title:
Demonstration of a Cas9 mRNA-mediated rescue of SP-B def. mice after i.t. delivery; detailed analyses of Cas9 mRNA-mediated gene editing in lungs of two different BL6 mouse strains after i.t. delivery
Duration:
01/08/2016 to 31/10/2016
Abstract / short description:
CureVac will design and produce mRNA encoding S. pyogenes cas9 and test the mRNA in vitro for functionality.

CureVac will provide 3 mg of cas9 mRNA to Prof. Kormann.

Prof. Kormann will use CureVac's cas9 mRNA to induce and analyze gene conversion in his surfactant protein B model as described in Mahiny et al., 2015 (Nat. Biotechnol. 33, 584-6), administering different mRNA doses. The required gRNA will be designed and ordered by Prof. Kormann.

Prof. Kormann will use CureVac's mRNA to induce and analyze allele conversion in two BL6 mouse strains, administering different mRNA doses. The required gRNA and ssODNs will be designed and ordered by Prof. Kormann. He will analyze frequency and correctness of conversion as well as frequency and identitiy of off-target effects.

Prof. Kormann will share the sequences of the finally selected gRNA(s) and ssODN(s).

Data will be shared between parties after completion of each experiment.
Keywords:
nec-mRNA
gene correction
gene therapy
Gentherapie
CFTR

Involved staff

Managers

Faculty of Medicine
University of Tübingen

Local organizational units

University Children’s Hospital - Department of Paediatrics
Hospitals and clinical institutes
Faculty of Medicine

Funders

Tübingen, Baden-Württemberg, Germany
Tübingen, Baden-Württemberg, Germany
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