Results 101 to 110 of about 21,845 (197)

A combinatorial oligonucleotide therapy to improve dystrophin restoration and dystrophin-deficient muscle health

Molecular Therapy: Nucleic Acids
Despite the proven safety of dystrophin-targeting phosphorodiamidate morpholino oligomer (PMO) therapy, poor delivery of the PMOs limit the efficacy of this dystrophin restoring gene therapy for Duchenne muscular dystrophy (DMD).
Young Jae Moon, Ravi Hindupur, Iteoluwakishi H. Gamu, Nikki M. McCormack, Fatima Shaikh, James S. Novak, Jyoti K. Jaiswal   +6 more
doaj   +1 more source

Immunologic investigations into transgene directed immune-mediated myositis following delandistrogene moxeparvovec gene therapy

Scientific Reports
Delandistrogene moxeparvovec is an rAAVrh74 vector-based gene transfer therapy that delivers a transgene encoding delandistrogene moxeparvovec micro-dystrophin, an engineered, functional form of dystrophin shown to stabilize or slow disease progression ...
Rachael A. Potter, Ida H. Moeller, Sohrab Khan, Hélène Haegel, Andreas Hollenstein, Guido Steiner, Christoph Wandel, Alexander P. Murphy, Damon R. Asher, Emanuel Palatinsky, Danielle A. Griffin, Stefanie Mason, Susan T. Iannaccone, Craig M. Zaidman, Louise R. Rodino-Klapac   +14 more
doaj   +1 more source

Engineering suppressor tRNAs for effective treatment of Duchenne muscular dystrophy. [PDF]

Sci Adv
Oury J, Chen YH, McFarland S, Jewel D, Wen J, Pasricha T, Villaseñor S, Mannheim W, Rose V, Zheng S, Thanigai Arasu GS, Calhoun M, Dilts A, Montminy M, Strozewski N, Thakur N, Kaplan PL, Eimon PM, Coller J, Lodish H.   +19 more
europepmc   +1 more source

Magnetic Mitohormesis as a Potential Non-Invasive Restorative Therapy for X-Linked Muscular Dystrophies. [PDF]

Int J Mol Sci
Iversen JN, Franco-Obregón A.
europepmc   +1 more source

Four new mouse models of Duchenne muscular dystrophy with clinically relevant exon deletions in the human DMD gene. [PDF]

Dis Model Mech
van Putten M, Linssen M, Tanganyika-de Winter C, Brouwers CM, Claassens JWC, Verwey N, Walsh M, Loredan Stan T, Aartsma-Rus A, Hohenstein P.   +9 more
europepmc   +1 more source

Dystrophin [PDF]

, 2020
openaire   +1 more source

Emerging Roles of Dystroglycan in Cardiac Remodeling, Fibrosis, and Heart Failure. [PDF]

Int J Mol Sci
Pradhan BS, Mączewski M.
europepmc   +1 more source

Duchenne muscular dystrophy: from gene to gene-ius therapies. [PDF]

Skelet Muscle
Muchir A, Musaro A, Chamberlain J.
europepmc   +1 more source

Bridging Duchenne muscular dystrophy therapy from rhesus monkeys to patients. [PDF]

Clin Transl Med
Guo W, Ji W, Chen Y.
europepmc   +1 more source

Prime Editing Mediated Generation and Correction of the mdx5cv Mutation Restores Dystrophin Expression in Myoblasts. [PDF]

Int J Mol Sci
Siddika A, Husseiny FE, Rousseau J, Tremblay JP.   +3 more
europepmc   +1 more source