Unveiling sequence-agnostic mixed-chemical modification patterns for splice-switching oligonucleotides using the NATURA platform
Chemical optimization of ribose has significantly advanced nucleic acid therapeutics (NATs)
by improving the stability, specificity, and safety of therapies like small interfering RNAs …
by improving the stability, specificity, and safety of therapies like small interfering RNAs …
Context-specific stress causes compartmentalized SARM1 activation and local degeneration in cortical neurons
FI Hinz, CLM Villegas, JT Roberts, H Yao… - Journal of …, 2024 - Soc Neuroscience
Sterile alpha and TIR motif containing 1 (SARM1) is an inducible NADase that localizes to
mitochondria throughout neurons and senses metabolic changes that occur after injury …
mitochondria throughout neurons and senses metabolic changes that occur after injury …
RNA Splicing Mechanisms in Rare Disease
V Tse - 2024 - search.proquest.com
The accurate expression of human genes requires precursor messenger RNA (pre-mRNA)
splicing. Pre-mRNA splicing removes superfluous sequences (introns) from pre-mRNA and …
splicing. Pre-mRNA splicing removes superfluous sequences (introns) from pre-mRNA and …