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 …
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 …
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
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 …
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
Introduction: Duchenne muscular dystrophy (DMD) is an X-linked recessive neuromuscular
disorder that affects approximately one in 3500–5000 male births. Patients experience …
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 …
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 …
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 …
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 …
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 …
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
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 …
one of the most common and lethal human hereditary disorders. A novel therapeutic …