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

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

doi:10.1016/j.omtn.2021.05.015

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

Thomas Kocher (First author), Johannes Bischof (Co-author), Simone Alexandra Haas, Oliver March (Co-author), Bernadette Liemberger (Co-author), Stefan Hainzl (Co-author), Julia Illmer, Anna Hoog (Co-author), Katharina Muigg (Co-author), Heide-Marie Binder, Alfred Klausegger (Co-author), Dirk Strunk (Co-author), Johann Bauer (Co-author), Toni Cathomen, Ulrich Koller^* (Last author)

^*Corresponding author for this work

Research output: Contribution to journal › Original Article (Journal) › peer-review

14 Citations (Web of Science)

Original language	English
Pages (from-to)	237-250
Journal	Molecular Therapy - Nucleic Acids
Volume	25
DOIs	https://doi.org/10.1016/j.omtn.2021.05.015
Publication status	Published - 2021

Keywords

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

Access to Document

10.1016/j.omtn.2021.05.015

Cite this

Kocher, T., Bischof, J., Haas, S. A., March, O., Liemberger, B., Hainzl, S., Illmer, J., Hoog, A., Muigg, K., Binder, H.-M., Klausegger, A., Strunk, D., Bauer, J., Cathomen, T., & Koller, U. (2021). A non-viral and selection-free COL7A1 HDR approach with improved safety profile for dystrophic epidermolysis bullosa. Molecular Therapy - Nucleic Acids, 25, 237-250. https://doi.org/10.1016/j.omtn.2021.05.015

@article{04067099428d4acb9f9504664db6a4ed,

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

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

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

note = "Kocher; Bischof; March; Liemberger; Hainzl; Illmer: Klausegger; Bauer; Koller: EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology and Allergology, University Hospital of the Paracelsus Medical University Salzburg, 5020 Salzburg, Austria; Hoog; Muigg; Binder; Strnk: Cell Therapy Institute, SCI-TReCS, Paracelsus Medical University, 5020 Salzburg, Austria ",

year = "2021",

doi = "10.1016/j.omtn.2021.05.015",

language = "English",

volume = "25",

pages = "237--250",

journal = "Molecular Therapy - Nucleic Acids",

issn = "2162-2531",

}

Kocher, T , Bischof, J, Haas, SA, March, O, Liemberger, B, Hainzl, S , Illmer, J, Hoog, A, Muigg, K, Binder, H-M, Klausegger, A , Strunk, D , Bauer, J, Cathomen, T & Koller, U 2021, 'A non-viral and selection-free COL7A1 HDR approach with improved safety profile for dystrophic epidermolysis bullosa.', Molecular Therapy - Nucleic Acids, vol. 25, pp. 237-250. https://doi.org/10.1016/j.omtn.2021.05.015

TY - JOUR

T1 - A non-viral and selection-free COL7A1 HDR approach with improved safety profile for dystrophic epidermolysis bullosa.

AU - Kocher, Thomas

AU - Bischof, Johannes

AU - Haas, Simone Alexandra

AU - March, Oliver

AU - Liemberger, Bernadette

AU - Hainzl, Stefan

AU - Illmer, Julia

AU - Hoog, Anna

AU - Muigg, Katharina

AU - Binder, Heide-Marie

AU - Klausegger, Alfred

AU - Strunk, Dirk

AU - Bauer, Johann

AU - Cathomen, Toni

AU - Koller, Ulrich

N1 - Kocher; Bischof; March; Liemberger; Hainzl; Illmer: Klausegger; Bauer; Koller: EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology and Allergology, University Hospital of the Paracelsus Medical University Salzburg, 5020 Salzburg, Austria; Hoog; Muigg; Binder; Strnk: Cell Therapy Institute, SCI-TReCS, Paracelsus Medical University, 5020 Salzburg, Austria

PY - 2021

Y1 - 2021

KW - RETROVIRAL VECTORS

KW - GENE CORRECTION

KW - CAS9 NUCLEASE

KW - CRISPR CAS9

KW - STEM-CELLS

KW - COL7A1

KW - DNA

KW - MUTATION

KW - TRANSPLANTATION

KW - KERATINOCYTES

U2 - 10.1016/j.omtn.2021.05.015

DO - 10.1016/j.omtn.2021.05.015

M3 - Original Article (Journal)

C2 - 34458008

SN - 2162-2531

VL - 25

SP - 237

EP - 250

JO - Molecular Therapy - Nucleic Acids

JF - Molecular Therapy - Nucleic Acids

ER -