Alternative splicing (AS) of pre-mRNA plays a crucial role in tissue-specific gene regulation, with disease implications due to splicing defects. Predicting and manipulating AS can therefore uncover ...
RNA sequencing has emerged as a powerful supplement to DNA sequencing for Mendelian disease diagnosis, but clinical translation of diagnostic RNA-seq has not been widely achieved. Researchers at ...
Avidity Biosciences (RNA) announced positive del-zota topline data from the Phase 1/2 EXPLORE44 trial in people living with Duchenne muscular ...
RNA sequencing has emerged as a powerful supplement to DNA sequencing for Mendelian disease diagnosis, but clinical translation of diagnostic RNA-seq ...
Despite growing competition in the transthyretin amyloid cardiomyopathy (ATTR-CM) field, Alnylam is hoping its RNA ...
In December 2024, ProQR expanded its collaboration with the Rett Syndrome Research Trust, securing additional funding for a total of up to $9.2 million to support the advancement of AX-2402, an RNA ...
A pair of privately-held developers of editing-based therapies have some successes to show for their recent efforts to ...
BMO Capital initiated coverage of Avidity Biosciences (RNA) with an Outperform rating and $72 price target Avidity’s pipeline includes ...
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