Stargardt disease type 1 (STGD1) is an inherited retinal recessive disease caused by biallelic variants in the ABCA4 gene. One of the recurrent variants is located at the exon-intron junction of exon ...
The Massachusetts based biotech will now press ahead with human trials for its antisense oligonucleotide (ASO) therapy, ...
Children's Mercy Kansas City has achieved a significant advancement toward the treatment of rare genetic diseases through the ...
Sanofi has entered into a collaboration and license agreement with Alloy Therapeutics to develop an antisense oligonucleotide (ASO) therapy targeting a single, undisclosed central nervous system ...
The FDA has granted fast-track designation to an investigational antisense oligonucleotide to treat individuals with myotonic dystrophy type 1, or DM1, according to the manufacturer. In a press ...
A drug that halts an AIDS-related eye infection could be the first antisense therapy to reach the market. However, whether the drug can truly be called "antisense" depends on some specifics--such as ...
The earlier deal gave Sanofi a stake in Resalis Therapeutics, an Italian biotech that is advancing an antisense oligonucleotide that targets a noncoding RNA involved in obesity pathways.
An investigational higher dose of spinal muscular atrophy drug nusinersen gains attention as the FDA and European Medicines ...
The FDA is putting Atara’s active Investigational New Drug applications on hold due to manufacturing concerns at a ...
From synthetic biology and gene therapy to ... Scientists use short oligonucleotides as primers for polymerase chain reaction and next-generation sequencing, in DNA microarrays and fluorescence in ...
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