Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy

JN Robinson-Hamm, CA Gersbach - Human genetics, 2016 - Springer
Duchenne muscular dystrophy is one of the most common inherited genetic diseases and is
caused by mutations to the DMD gene that encodes the dystrophin protein. Recent …

Correction of three prominent mutations in mouse and human models of Duchenne muscular dystrophy by single-cut genome editing

YL Min, F Chemello, H Li, C Rodriguez-Caycedo… - Molecular Therapy, 2020 - cell.com
Duchenne muscular dystrophy (DMD), one of the most common neuromuscular disorders of
children, is caused by the absence of dystrophin protein in striated muscle. Deletions of …

Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy

L Amoasii, C Long, H Li, AA Mireault… - Science translational …, 2017 - science.org
Duchenne muscular dystrophy (DMD) is a severe, progressive muscle disease caused by
mutations in the dystrophin gene. The majority of DMD mutations are deletions that …

Restoration of dystrophin expression and correction of Duchenne muscular dystrophy by genome editing

T Aslesh, E Erkut, T Yokota - Expert Opinion on Biological Therapy, 2021 - Taylor & Francis
Introduction: Duchenne muscular dystrophy (DMD) is an X-linked recessive neuromuscular
disorder that affects approximately one in 3500–5000 male births. Patients experience …

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

CE Nelson, CH Hakim, DG Ousterout, PI Thakore… - Science, 2016 - science.org
Duchenne muscular dystrophy (DMD) is a devastating disease affecting about 1 out of 5000
male births and caused by mutations in the dystrophin gene. Genome editing has the …

A humanized knockin mouse model of Duchenne muscular dystrophy and its correction by CRISPR-Cas9 therapeutic gene editing

Y Zhang, H Li, T Nishiyama, JR McAnally… - … Therapy-Nucleic Acids, 2022 - cell.com
Duchenne muscular dystrophy (DMD) is a lethal neuromuscular disease caused by
mutations in the X-linked dystrophin (DMD) gene. Exon deletions flanking exon 51, which …

Restoration of dystrophin protein expression by exon skipping utilizing CRISPR-Cas9 in myoblasts derived from DMD patient iPS cells

M Ifuku, KA Iwabuchi, M Tanaka, MSY Lung… - Exon Skipping and …, 2018 - Springer
Duchenne muscular dystrophy (DMD) is a congenital X-linked disease caused by mutations
in the gene encoding the dystrophin protein, which is required for myofiber integrity. Exon …

[HTML][HTML] Promising therapeutic approaches using CRISPR/Cas9 genome editing technology in the treatment of Duchenne muscular dystrophy

H Mollanoori, Y Rahmati, B Hassani, MH Mehr… - Genes & …, 2021 - Elsevier
Duchenne muscular dystrophy is an X-linked recessive hereditary monogenic disorder
caused by inability to produce dystrophin protein. In most patients, the expression of …

Creation of a novel humanized dystrophic mouse model of Duchenne muscular dystrophy and application of a CRISPR/Cas9 gene editing therapy

CS Young, E Mokhonova, M Quinonez… - Journal of …, 2017 - content.iospress.com
Duchenne muscular dystrophy is caused by mutations in DMD which disrupt the reading
frame. Therapeutic strategies that restore DMD's reading frame, such as exon skipping and …

New advancements in CRISPR based gene therapy of Duchenne muscular dystrophy

A Eslahi, F Alizadeh, A Avan, GA Ferns, M Moghbeli… - Gene, 2023 - Elsevier
Duchenne muscular dystrophy (DMD) is caused by the dystrophin gene mutations and is
one of the most common and lethal human hereditary disorders. A novel therapeutic …