Marc Moore
Darlington, ENG DL11HG, United Kingdom
Last Updated: 09/26/2026
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Found 8 publications
Golodirsen restores DMD transcript imbalance in Duchenne Muscular Dystrophy patient muscle cells.
Journal: Skeletal muscle
Published: July 12, 2024
Restoration of Normal NF1 Function with Antisense Morpholino Treatment of Recurrent Pathogenic Patient-Specific Variant c.1466A>G; p.Y489C.
Journal: Journal of personalized medicine
Published: October 07, 2021
Targeted exon skipping of NF1 exon 17 as a therapeutic for neurofibromatosis type I.
Journal: Molecular therapy. Nucleic acids
Published: July 28, 2021
Optimized lentiviral vector to restore full-length dystrophin via a cell-mediated approach in a mouse model of Duchenne muscular dystrophy.
Journal: Molecular therapy. Methods & clinical development
Published: July 12, 2021
Re-structuring lentiviral vectors to express genomic RNA via cap-dependent translation.
Journal: Molecular therapy. Methods & clinical development
Published: March 06, 2020
Reversible immortalisation enables genetic correction of human muscle progenitors and engineering of next-generation human artificial chromosomes for Duchenne muscular dystrophy.
Journal: EMBO molecular medicine
Published: December 16, 2017
Current Progress in Therapeutic Gene Editing for Monogenic Diseases.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: October 05, 2015
Showing 1-8 of 8
Last Updated: 09/26/2026