ProQR Therapeutics is advancing its Axiomer ADAR-mediated RNA editing pipeline, aiming to file a Clinical Trial Application for its lead program AX-0810, targeting NTCP for cholestatic diseases, by Q2 ...

A research team led by Rice's Yang Gao has uncovered new insights into the molecular mechanisms of ADAR1. ADAR1 converts adenosine to inosine in double-stranded RNA, a process essential for preventing ...

Parkinson's disease (PD) is a neurodegenerative disorder in which the α-synuclein protein abnormally aggregates within brain cells, causing neuronal damage. Through international collaboration, ...

Wave expects to initiate a Phase 2a multidose portion of WVE-007 INLIGHT clinical trial in individuals living with obesity ...

Korro Bio's RNA editing platform, OPERA, targets genetic diseases by performing precise, transient single-base edits on RNA, reducing the risks associated with DNA editing. Lead asset KRRO-110 aims to ...

Advances in the gene-editing technology known as CRISPR-Cas9 over the past 15 years have yielded important new insights into the roles that specific genes play in many diseases. But to date this ...

LEIDEN, Netherlands & CAMBRIDGE, Mass., Nov. 06, 2023 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today ...

All starting from the same DNA, neurons ultimately take on individual characteristics in the brain and body. Differences in which genes they transcribe into RNA help determine which type of neuron ...

An ancient RNA-guided system with potential to expand gene-editing capabilities has been identified. Researchers at MIT’s McGovern Institute and the Broad Institute of MIT and Harvard (both MA, USA) ...

Genetic editing holds promise to treat incurable diseases, but the most popular method - CRISPR - sometimes does more harm than good. A new study from University of California San Diego and Yale ...