University of British Columbia and Vancouver Coastal Health scientists have developed a potential cure for a rare eye disease, showing for the first time that a drug can repair a birth defect.
Dec 20, 2013
0
0
A study published today by scientists at University of Massachusetts Medical School and the University of Alabama at Birmingham provides insight into the mechanism of action of the drug ataluren, which is showing promise ...
Oct 4, 2016
0
1
In a study published today in Nature, Marc Bühler and his group at the Friedrich Miescher Institute for Biomedical Research (FMI) have taken a major step forward in elucidating the mechanisms underlying a disorder known ...
May 24, 2018
0
57
Usher syndrome is the most common form of combined congenital deaf-blindness in humans and affects 1 in 6,000 of the population. It is a recessive inherited disease that is both clinically and genetically heterogeneous. In ...
Jul 1, 2011
0
0
Scientists at the Institute for Research in Biomedicine (IRB Barcelona), in collaboration with the Centre for Genomic Regulation (CRG) and Radboud University, have developed an algorithm that can predict which cancer patients ...
Oct 30, 2019
0
29
Cancer tumours manipulate a natural cell process to promote their survival suggesting that controlling this mechanism could stop progress of the disease, according to new research led by the University of Oxford.
Jun 26, 2017
0
7
A multi-institutional research group has succeeded in developing an exon-skipping therapy using nucleic-acid therapeutics for Alport syndrome, an incurable kidney disease that can progress to renal failure. Disease model ...
Jun 25, 2020
0
2
Nonsense-mediated RNA decay, or NMD, is an evolutionarily conserved molecular mechanism in which potentially defective messenger RNAs, or mRNAs, are degraded. By reducing errors in gene expression, it serves as an RNA quality ...
Feb 3, 2022
0
18
An experimental drug reported in Nature Communications suggests that a "path is clearly achievable" to treat currently untreatable cases of cystic fibrosis disease caused by nonsense mutations. This includes about 11 percent ...
Jul 19, 2021
0
66
A drug developed to treat genetic diseases such as Duchenne muscular dystrophy and cystic fibrosis may need a radical rethink. In a new study published on 25 June in the open access journal PLOS Biology, researchers question ...
Jun 25, 2013
0
0
