Root causal genes correspond to the initial changes to gene expression that induce a diagnosis and can be reliably detected from bulk RNA-seq after determining a causal ordering from Perturb-seq.
An optimized cytosine base editing system for Leishmania species improves editing efficiency, reduces toxicity, and ensures stable guide expression, enabling scalable loss-of-function screening and efficient creation of functional mutants.
Monique Marylin Alves de Almeida, Yves De Repentigny ... Rashmi Kothary
A liver-specific depletion of the survival motor neuron protein in a mouse model unveils a compelling and intricate connection between liver function and pancreatic abnormalities in spinal muscular atrophy.
The UA-dinucleotide ratio in UTRs is negatively correlated with RNA stability both in the massively parallel reporter assay and in vivo, and is prevalent in fast-turnover genes.
In P3 editing, molecular interactions are sensed and converted directly into genome editing events, which may be useful for biological recording and synthetic molecular circuits deployed in living cells.