Results 191 to 200 of about 56,717 (273)

A Novel Transcriptional Slippage Mechanism Rescues Dystrophin Expression from a DMD Frameshift Variant. [PDF]

Ann Neurol
Naruse H, Mitsui J, Kubota A, Okubo S, Mitsuchi S, Sumi K, Tanifuji S, Komaki S, Kitamura A, Maeda M, Yashita D, Sudo A, Matsukawa T, Hamada M, Satake W, Tsuji S, Toda T.   +16 more
europepmc   +1 more source

Genetic Mutations Underlying Growth Impairment and Cardiomyopathies in Children: Molecular Mechanisms, Clinical Implications and Targeted Therapies. [PDF]

Genes (Basel)
Dicorato MM, De Sario G, Carella MC, Guaricci AI, Ciccone MM, Forleo C, D'Amato G, Faienza MF.   +7 more
europepmc   +1 more source

Functional disruption of the dystrophin gene in rhesus monkey using CRISPR/Cas9.

Human Molecular Genetics, 2015
Yongchang Chen, Yinghui Zheng, Yu Kang, Weili Yang, Yuyu Niu, Xiangyu Guo, Zhuchi Tu, Chenyang Si, Hong Wang, Ruxiao Xing, X. Pu, Shang-Hsun Yang, Shihua Li, W. Ji, Xiao-Jiang Li   +14 more
semanticscholar   +1 more source

A new dystrophin-deficient rat model mirroring exon skipping in patients with DMD exon 45 deletions. [PDF]

Dis Model Mech
Wang T, Daoud C, Dubois A, Corre G, Bellec J, Bovolenta M, Philidet L, Dorval A, Bourg N, Roudaut C, Albini S, Warthi G, Jaber A, Richard I.   +13 more
europepmc   +1 more source

Monoclonal antibodies recognizing dystrophin and dystrophin-associated protein A1.

Japanese Journal of Pharmacology, 1991
Yasuko Hagiwara, Mikiharu Yoshida, Eijiro Ozawa   +2 more
openaire   +1 more source

Dystrophin Restorative and Compensatory Gene Addition Therapies for Duchenne Muscular Dystrophy: Could CRISPRa Provide a Realistic Alternative? [PDF]

Muscles
Rostamitehrani Z, Javed R, Popplewell L.
europepmc   +1 more source

Genetic strategies for therapy of Duchenne muscular dystrophy. [PDF]

Mol Ther Nucleic Acids
Łoboda A, Chamberlain JS, Dulak J.
europepmc   +1 more source

Impact of dystrophin deficiency on vascular smooth muscle cell. [PDF]

Sci Rep
Xuan W, Cheng F, Han X, Tipparaju SM, Ashraf M.   +4 more
europepmc   +1 more source

Deaths in gene therapy of Duchenne muscular dystrophy and other diseases: Underlying mechanisms and mitigating strategies. [PDF]

Mol Ther
Duan D, Herzog RW.
europepmc   +1 more source

Histone deacetylases in Duchenne muscular dystrophy: a role in the mechanism of disease and a target for inhibition. [PDF]

Clin Epigenetics
Bertoldi M, Albamonte E, Bello L, D'amico A, Masson R, Nigro V, Pane M, Panicucci C, Sframeli M, Ricci F.   +9 more
europepmc   +1 more source