Paracelsus Medizinische Privatuniversität (PMU)

Research & Innovation
Publications

A non-viral and selection-free COL7A1 HDR approach with improved safety profile for dystrophic epidermolysis bullosa.

#2021
#Molecular Therapy - Nucleic Acids

PMU Authors
Thomas Kocher, Johannes Bischof, Oliver March, Bernadette Liemberger, Stefan Hainzl, Julia Illmer, Anna Hoog, Katharina Muigg, Alfred Klausegger, Dirk Strunk, Johann Bauer, Ulrich Koller

All Authors
Thomas Kocher, Johannes Bischof, Simone Alexandra Haas, Oliver March, Bernadette Liemberger, Stefan Hainzl, Julia Illmer, Anna Hoog, Katharina Muigg, Heide-Marie Binder, Alfred Klausegger, Dirk Strunk, Johann Bauer, Toni Cathomen, Ulrich Koller

Journal association
Molecular Therapy - Nucleic Acids

Abstract

Keywords

TRANSPLANTATION, STEM-CELLS, KERATINOCYTES, DNA, MUTATION, COL7A1, RETROVIRAL VECTORS, GENE CORRECTION, CRISPR CAS9, CAS9 NUCLEASE