BioCentury | Dec 6, 2018
Translation in Brief

New Therapeutic Targets and Biomarkers: November 2018

...caused by gain-of-function mutations in the gene Duke University School of Medicine Distillery Therapeutics Retinitis Pre-RNA processing factor 31 (PRPF31)...
BioCentury | Nov 6, 2018
Distillery Therapeutics

Ophthalmic disease

...gene editing therapy in animal models of PRPF31-mutant retinitis pigmentosa. TARGET/MARKER/PATHWAY: CRISPR-associated protein 9 (Cas9); pre-RNA processing factor 31 (PRPF31)...
...CONTACT: Majlinda Lako, Newcastle University, Newcastle upon Tyne, U.K. email: majlinda.lako@ncl.ac.uk Jaime De Leon Newcastle University University of Leeds Pre-RNA processing factor 31 (PRPF31) Retinitis...
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