Alternative splicing enables a single gene to encode multiple mRNA variants and, consequently, diversifies protein isoform products. The advent of deep sequencing technologies has revealed that 95 ...

RNA splicing involves the removal of non-coding introns and the joining of coding exons in precursor mRNA (pre-mRNA) to produce mature messenger RNA (mRNA). Splicing changes can lead to diseases ...

Ribopeutic Inc. has divulged nucleic acid splicing modulators (particularly, Huntingtin [HTT; HD] [mutant] and/or transcriptional activator Myb [c-Myb]) reported to be useful for the treatment of ...

Skyhawk Therapeutics Announces Positive Topline Results from Parts A and B of its Phase 1 Clinical Trial of SKY-0515 as a Treatment for Huntington’s Disease, Reaching 72% Huntingtin mRNA ...

This project aims to develop mRNA anti-cancer vaccines based on shared frequently misspliced aberrations and test whether these are enhanced in combination with other therapies. Project aims Aim 1: ...

Novartis NVS announced that the FDA has granted orphan drug designation to branaplam (LMI070) in Huntington’s disease (HD).Huntington’s disease is a rare, inherited neurodegenerative disease ...

In Huntington’s disease, the mutant huntingtin gene (HTT) mRNA is retained in the nucleus and forms insoluble clusters. A new study shows that short interfering RNA (siRNA), an oligonucleotide ...

Skyhawk's SKY-0515 demonstrated dose-dependent huntingtin (HTT) mRNA reduction in healthy volunteers, with 72% reduction at the highest dose tested in the multiple ascending dose study SKY-0515 was ...

Skyhawk Therapeutics Announces Positive Topline Results from Parts A and B of its Phase 1 Clinical Trial of SKY-0515 as a Treatment for Huntington’s Disease, Reaching 72% Huntingtin mRNA Reduction ...