Results 91 to 100 of about 114,439 (345)
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
Implantable Drug Delivery Systems for Skeletal Muscles and Eyes
Advanced NanoBiomed Research, EarlyView.This review highlights the different types of recent implantable drug delivery systems (IDDS) fabricated for a use with skeletal muscles, and with eyes. It presents the developments already made and the current research directions, showing the evolution of IDDS and their great diversity.
Serge Ostrovidov +8 more
wiley +1 more source
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
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
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
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
Contrasting Experimentally Device-Manipulated and Device-Free Smiles. [PDF]
, 2019 Researchers in psychology have long been interested in not only studying smiles, but in examining the downstream effects of experimentally manipulated smiles.
Cross, Marie P +2 more
core
Performance of Upper Limb module for Duchenne muscular dystrophy
Developmental Medicine & Child Neurology, 2020 To report the differences between Performance of Upper Limb (PUL) versions 1.2 and 2.0, compare the measurement ability of the two versions, and compare their longitudinal performance in Duchenne muscular dystrophy.
A. Mayhew +8 more
semanticscholar +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