New and emerging pharmacotherapy for duchenne muscular dystrophy: A focus on synthetic therapeutics
SM Grages, M Bell, DJ Berlau - Expert Opinion on …, 2020 - Taylor & Francis
Introduction Duchenne muscular dystrophy (DMD) is the result of X-chromosome-linked
mutations to the dystrophin protein gene that prevent the normal development and repair of …
mutations to the dystrophin protein gene that prevent the normal development and repair of …
Current and emerging treatment strategies for Duchenne muscular dystrophy
JK Mah - Neuropsychiatric disease and treatment, 2016 - Taylor & Francis
Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy in
childhood. It is caused by mutations of the DMD gene, leading to progressive muscle …
childhood. It is caused by mutations of the DMD gene, leading to progressive muscle …
Duchenne muscular dystrophy: pathogenesis and promising therapies
M Chang, Y Cai, Z Gao, X Chen, B Liu, C Zhang… - Journal of …, 2023 - Springer
Duchenne muscular dystrophy (DMD) is a severe, progressive, muscle-wasting disease,
characterized by progressive deterioration of skeletal muscle that causes rapid loss of …
characterized by progressive deterioration of skeletal muscle that causes rapid loss of …
[图书][B] Duchenne muscular dystrophy: advances in therapeutics
JS Chamberlain, TA Rando - 2006 - books.google.com
Duchenne Muscular Dystrophy (DMD) is one of the most prevalent genetic disorders of
childhood for which there is no cure. This authoritative guide provides a clear overview of …
childhood for which there is no cure. This authoritative guide provides a clear overview of …
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 …
Exon-skipping in Duchenne muscular dystrophy
S Takeda, PR Clemens… - Journal of neuromuscular …, 2021 - content.iospress.com
Duchenne muscular dystrophy (DMD) is a devastating, rare disease. While clinically
described in the 19 th century, the genetic foundation of DMD was not discovered until more …
described in the 19 th century, the genetic foundation of DMD was not discovered until more …
Clinical manifestations and overall management strategies for Duchenne muscular dystrophy
T Tsuda - Duchenne Muscular Dystrophy: Methods and Protocols, 2018 - Springer
Duchenne muscular dystrophy (DMD) is an X-linked genetic disorder that causes
progressive weakness and wasting of skeletal muscular and myocardium in boys due to …
progressive weakness and wasting of skeletal muscular and myocardium in boys due to …
Duchenne muscular dystrophy: Current treatment and emerging exon skipping and gene therapy approach
G Patterson, H Conner, M Groneman, C Blavo… - European Journal of …, 2023 - Elsevier
Duchenne muscular dystrophy (DMD) is an X-linked recessive neuromuscular disorder that
causes debilitating muscle weakness and atrophy due to a loss of the dystrophin protein …
causes debilitating muscle weakness and atrophy due to a loss of the dystrophin protein …
DMD Mutations in 576 Dystrophinopathy Families: A Step Forward in Genotype-Phenotype Correlations
Recent advances in molecular therapies for Duchenne muscular dystrophy (DMD) require
precise genetic diagnosis because most therapeutic strategies are mutation-specific. To …
precise genetic diagnosis because most therapeutic strategies are mutation-specific. To …
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 …