Results 61 to 70 of about 71,442 (257)
F1000Research, 2016 Deformation imaging by echocardiography is a well-established research tool which has been gaining interest from clinical cardiologists since the introduction of speckle tracking.
Oana Mirea +2 more
doaj +1 more source
Biological Reviews, EarlyView.ABSTRACT Three categories of explanations exist for why we age: mechanistic theories, which omit reference to evolutionary forces; weakening force of selection theories, which posit that barriers exist that prevent evolutionary forces from optimising fitness in ageing; and optimisation theories, which posit that evolutionary forces actually select for ...
Michael S. Ringel
wiley +1 more source
CLINICAL CASE OF FAMILIAL DUCHENNE MYODYSTROPHY
Мать и дитя в Кузбассе, 2022 The article presents an analysis of a clinical case of the diagnosis of familial Duchenne myodystrophy in a newborn child confirmed in the neonatal period. The stages of diagnosis and their peculiarities are described.
Марина Афанасьевна Соколовская +2 more
doaj
International Journal of Neonatal Screening, 2019 Duchenne muscular dystrophy (DMD/Duchenne) is a progressive X-linked disease and is the most common pediatric-onset form of muscular dystrophy, affecting approximately 1:5000 live male births. DNA testing for mutations in the dystrophin gene confirms the
Anne Timonen +11 more
doaj +1 more source
Long-term Evaluation of AAV-CRISPR Genome Editing for Duchenne Muscular Dystrophy
Nature Medicine, 2018 Duchenne muscular dystrophy (DMD) is a monogenic disorder and a candidate for therapeutic genome editing. There have been several recent reports of genome editing in preclinical models of Duchenne muscular dystrophy1–6, however, the long-term persistence
Christopher E. Nelson +11 more
semanticscholar +1 more source
Viltolarsen in Japanese Duchenne muscular dystrophy patients: A phase 1/2 study
Annals of Clinical and Translational Neurology, 2020 The novel morpholino antisense oligonucleotide viltolarsen targets exon 53 of the dystrophin gene, and could be an effective treatment for patients with Duchenne muscular dystrophy (DMD).
H. Komaki +11 more
semanticscholar +1 more source
Optimization of CEST MRI Reporter Protein Design Using Cation‐Pi Networks
Chemistry – A European Journal, EarlyView.A novel engineering approach can produce reporter proteins for cell and viral therapy tracking with unique magnetic resonance imaging (MRI) signatures, detectable with chemical exchange saturation transfer (CEST). We discover how cation‐π interactions between amino acid groups can help us fine‐tune magnetic resonance properties for noninvasive ...
David E. Korenchan +8 more
wiley +1 more source
Fhod3 in zebrafish supports myofibril stability during growth of embryonic skeletal muscle
Developmental Dynamics, EarlyView.Abstract Background Actin filament organization in cardiomyocytes critically depends on the formin Fhod3, but a role for Fhod3 in skeletal muscle development has not yet been described. Results We demonstrate here that in zebrafish mutated for one of two fhod3 paralog genes, fhod3a, skeletal muscle of the trunk appears normal through 2 days post ...
Aubrie Russell +3 more
wiley +1 more source
Health and Quality of Life Outcomes, 2021 Background In clinical trials for rare diseases, such as Duchenne muscular dystrophy, clinical outcome assessments (COA) used to assess treatment benefit are often generic and may not be sensitive enough to detect change in specific patient populations ...
Hannah Staunton +7 more
doaj +1 more source
CRISPR Correction of Duchenne Muscular Dystrophy.
Annual Review of Medicine, 2019 The ability to efficiently modify the genome using CRISPR technology has rapidly revolutionized biology and genetics and will soon transform medicine.
Yi-Li Min, R. Bassel-Duby, E. Olson
semanticscholar +1 more source