Retinitis pigmentosa (RP) is a genetic eye disorder affecting around one in 5,000 people worldwide. It typically begins with night blindness in youth and progresses to tunnel vision as ...
Data demonstrating superior selectivity, safety and pharmacokinetics of RGT-0474060 were presented at the 21st Annual Huntington’s Disease ...
Researchers show two distinct MECP2 mutations in Rett syndrome elevate miRNA-126-3p, lowering ZO-1 and weakening blood-brain ...
Neuroscientists discover that Rett syndrome mutations cause a leaky brain via overexpressed miRNA-126-3p, pointing to a new therapeutic target for vascular repair.
Researchers discover that the RNA surveillance system NMD and the protein UPF2 are essential for guiding neuronal migration and forming the brain's layered cortex.
Morning Overview on MSN
RNA strand that nearly self-replicates could unlock life’s origin
A 45-nucleotide strand of RNA can now synthesize both its mirror-image complement and a copy of itself, bringing scientists ...
Thousands of previously “invisible” microproteins—tiny chains of fewer than 100 amino acids—can profoundly change human biology when mutated. A fundamental discovery is overturning decades of ...
Dr. Anczukow is a leading cancer biologist with expertise in RNA biology, molecular and cell biology, as well as human genetics, cancer biology, and genomics Olga Anczukow, Ph.D. Olga Anczukow, Ph.D.
How innovative technologies like AI and gene editing are advancing rare disease drug discovery and development.
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