Scientists at St. Jude Children's Research Hospital have found that they can reverse the effects of HNRNPH2-related ...
The pivotal study of zilganersen in Alexander disease missed a secondary endpoint, but analysts expect the FDA to approve the ...
Biogen has agreed to acquire Apellis Pharmaceuticals after an extended negotiation process. The company also entered a collaboration with Alloy Therapeutics focused on antisense drug development using ...
After a two-decade cycle of investor embrace and rejection, partnerships won and lost, failed clinical trials, layoffs, and rebuilds, Isis Pharmaceuticals has gained approval for the first ...
Following the initial reports and characterization of gene silencing through endogenous and exogenous antisense RNAs, the first antisense oligonucleotide drug, fomivirsen, received regulatory approval ...
While studying Parkinson's disease, an international research group made a discovery which can improve industrial protein synthesis for therapeutic use. They managed to understand a novel function of ...
Frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS) are two fatal neurodegenerative diseases marked by different clinical signs. But they are believed to be caused by a common ...
The hexanucleotide repeats in the C9ORF72 gene that cause amyotrophic lateral sclerosis and frontotemporal dementia undergo transcription and translation in both the sense and antisense directions.
An antisense therapy restores production of the protein FMRP in cell samples taken from patients with fragile X syndrome. This breakthrough was possible because of the novel findings, also presented ...
Johnson & Johnson’s Janssen Biotech will use Isis Pharmaceuticals’ RNA-targeted technology platform to discover and develop antisense drugs to treat autoimmune disorders of the gastrointestinal (GI) ...
The puzzle of neurodegenerative disease caused by expansions in the C9ORF72 gene just grew more intricate. In four recent papers, researchers report that the repeat is transcribed and translated not ...
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