Enhanced HDR-mediated correction of heterozygous COL7A1 mutations for recessive dystrophic epidermolysis bullosa.

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Title: Enhanced HDR-mediated correction of heterozygous COL7A1 mutations for recessive dystrophic epidermolysis bullosa.
Authors: Hunt JMT; School of Biological Sciences, The University of Auckland, Auckland 1010, New Zealand.; Maurice Wilkins Center, New Zealand., du Rand A; School of Biological Sciences, The University of Auckland, Auckland 1010, New Zealand., Verdon D; School of Biological Sciences, The University of Auckland, Auckland 1010, New Zealand., Clemance L; School of Biological Sciences, The University of Auckland, Auckland 1010, New Zealand., Loef E; School of Biological Sciences, The University of Auckland, Auckland 1010, New Zealand., Malhi C; School of Biological Sciences, The University of Auckland, Auckland 1010, New Zealand., Buttle B; School of Biological Sciences, The University of Auckland, Auckland 1010, New Zealand., Knapp DJHF; Institut de recherche en immunologie et en cancérologie (IRIC) and Département de Pathologie et Biologie Cellulaire, Université de Montréal, Montréal, QC H3T 1J4, Canada., Michaels YS; Paul Albrechtsen Research Institute CancerCare Manitoba, Winnipeg, MB R2H 2A6, Canada.; Department of Biochemistry and Medical Genetics, Rady Faculty of Health Sciences, University of Manitoba, Winnipeg, MB R3E 0J9, Canada., Garlick J; School of Dental Medicine, Tufts University, Boston, MA 02111, USA., Dunbar PR; School of Biological Sciences, The University of Auckland, Auckland 1010, New Zealand.; Maurice Wilkins Center, New Zealand., Purvis D; Te Whatu Ora Health New Zealand, Te Toka Tumai, Auckland 1023, New Zealand., Feisst V; School of Biological Sciences, The University of Auckland, Auckland 1010, New Zealand., Sheppard H; School of Biological Sciences, The University of Auckland, Auckland 1010, New Zealand.; Maurice Wilkins Center, New Zealand.
Source: Molecular therapy. Nucleic acids [Mol Ther Nucleic Acids] 2025 Feb 01; Vol. 36 (1), pp. 102472. Date of Electronic Publication: 2025 Feb 01 (Print Publication: 2025).
Publication Type: Journal Article
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 101581621 Publication Model: eCollection Cited Medium: Print ISSN: 2162-2531 (Print) Linking ISSN: 21622531 NLM ISO Abbreviation: Mol Ther Nucleic Acids Subsets: PubMed not MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:2162-2531
DOI:10.1016/j.omtn.2025.102472