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, ...

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 ...

LEIDEN, Netherlands & CAMBRIDGE, Mass., June 18, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV (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 ...

2026年1月2日，北京大学生命科学学院伊成器教授课题组在Nature Biotechnology在线发表研究论文。该研究建立了由单链脱氨酶辅助的可调节RNA信息操纵平台AIM（adjustable RNA information manipulation）。该平台突破了目前RNA碱基编辑工具“单点编辑”的限制，实现了在RNA靶向区域内对不同数量、多种类型碱基的可 ...

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

In a paper published on aBIOTECH, the authors investigated the off-target effects of ADAR-mediated RNA editing in rice ...

Initial AX-0810 data show no safety signals after 4 weeks of dosing and pharmacokinetics consistent with non-clinical data; ...

Treating acute myeloid leukemia (AML) depends on knowing what goes wrong inside cells. A new study suggests that two genetic ...