Paracelsus Medizinische Privatuniversität (PMU)

Forschung & Innovation
Publikationen

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

#2021
#Molecular Therapy - Nucleic Acids

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

Alle Autor*innen
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

Fachzeitschrift
Molecular Therapy - Nucleic Acids

Kurzfassung

Keywords

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