超越CRISPR：基于ADAR的RNA编辑技术，正在重塑癌症等疾病的治疗

Core Insights - The ADAR (Adenosine deaminases acting on RNA) family plays a crucial role in RNA editing, immune homeostasis, and neurological functions, with significant advancements in ADAR-mediated RNA editing technology in recent years [2] - Unlike CRISPR-based editing, ADAR-based RNA editing requires only the expression of endogenous deaminase ADAR in cells, avoiding delivery challenges and related immunogenicity issues, thus providing a safer option for precise RNA editing without altering genomic sequences [2] - ADAR-mediated RNA editing shows great potential in treating genetic diseases and cancers, with clinical trials approved for therapies targeting conditions like α-1 antitrypsin deficiency (AATD) and Stargardt disease, as well as inhibiting tumor growth by precisely editing oncogenic mRNA [2] Group 1: ADAR Family and RNA Editing - The ADAR family is essential for catalyzing RNA A-to-I editing, which regulates RNA diversity and maintains immune and neurological functions [2] - ADAR-mediated RNA editing technology has rapidly developed, distinguishing itself from CRISPR-based methods by its safety and precision [2] Group 2: ADAR1 as a Drug Target - ADAR1 is closely associated with various diseases, including type I interferonopathies, cancers, and viral infections, making it an important drug target [3] - Development strategies for ADAR1 inhibitors include targeting its catalytic domain, regulating upstream and downstream signaling pathways, and employing PROTAC degradation technology, showing promise in cancer and autoimmune disease treatments [3] Group 3: Upcoming Webinar - A webinar titled "Decoding ADAR: From RNA Editing, Innovative Inhibitor Development Strategies to SignalChem Screening Services" is scheduled for November 20, featuring experts from the industry [4][8] - Judy Zhu, a senior product manager, will discuss the role of ADAR-mediated RNA editing in cancer treatment and the significance of ADAR1 as a drug target [3][10] - Eric Yao, a senior scientist, will present on the development and screening strategies for ADAR1-targeted inhibitors, highlighting SignalChem's services in supporting research and drug development [3][9]