The initial clinical trial of a novel approach to treating amyotrophic lateral sclerosis (ALS) – blocking production of a mutant protein that causes an inherited form of the progressive neurodegererative disease – may ...
Apr 3, 2013
0
0
Non-alcoholic fatty liver disease (NAFLD) is a major health problem affecting approximately 30% of people in Western countries and which has been in sharp increase over the last three decades.
Dec 5, 2023
0
2
Duchenne muscular dystrophy occurs in boys and is characterized by progressive muscle degeneration and weakness leading to a decline in respiratory function. Strategies to arrest this severe progressive deterioration are ...
Jul 9, 2019
0
0
A team of scientists, comprising researchers from the Institute of Molecular and Cell Biology (IMCB), a research institute under the Agency for Science, Technology and Research (A*STAR), Singapore, and the VIB Laboratory ...
Dec 16, 2015
0
3
The dystrophin protein offers critical support to muscle fibers. Mutations affecting dystrophin's expression cause the muscle-wasting disease muscular dystrophy. In Duchenne muscular dystrophy (DMD), these mutations take ...
Jan 30, 2013
0
0
In a proof-of-concept study, researchers demonstrated the effectiveness of a potential new therapy for Timothy syndrome, an often life-threatening and rare genetic disorder that affects a wide range of bodily systems, leading ...
Apr 24, 2024
0
27
Bacteria's internal bomb, the so-called toxin-antitoxin (TA) system that is part of the normal bacterial makeup, may be triggered to make bacteria turn on themselves, providing a valuable target for novel antimicrobial approaches ...
Apr 21, 2018
0
2
The development of hepatocellular carcinoma, liver cancer, is known to involve the abnormal activation of a signaling pathway called Wnt; however, the underlying mechanisms have not been fully understood, until now.
Jun 28, 2023
0
3
Adding exercise to a genetic treatment for myotonic dystrophy type 1 (DM1) was more effective at reversing fatigue than administering the treatment alone in a study using a mouse model of the disease. In fact, exercise alone ...
Nov 30, 2020
0
8
Skoltech researchers and their colleagues from Russia and the UK investigated the safety and efficacy of new chemistry in antisense oligonucleotides used to treat spinal muscular atrophy (SMA), a debilitating genetic disease. ...
Jun 29, 2021
0
3
