基于ADAR的RNA编辑疗法
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超越CRISPR:基于ADAR的RNA编辑技术,正在重塑癌症等疾病的治疗
生物世界· 2025-11-14 10:00
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 - ADAR1 is closely associated with various diseases, including type I interferon diseases, cancer, and viral infections, making it an important drug target [3] - Development strategies for ADAR1 inhibitors are diverse, including targeting its catalytic domain, regulating upstream and downstream signaling pathways, and employing PROTAC degradation technology, showing broad prospects in cancer and autoimmune disease treatment [3] - A live lecture titled "Decoding ADAR: From RNA Editing, Innovative Inhibitor Development Strategies to SignalChem Screening Services" was held on November 20, featuring experts discussing the role of ADAR-mediated RNA editing in cancer treatment and the significance of ADAR1 as a drug target [3][4] Group 2 - The lecture included key topics such as the structural characteristics and biological functions of the ADAR family, applications of ADAR-mediated RNA editing in cancer therapy, the relationship between ADAR1 and diseases, and its importance as a drug target [8][12] - Strategies and methods for developing ADAR1-targeted inhibitors and SignalChem's ADAR inhibitor screening services were also discussed, highlighting their systematic support for research and new drug development in related disease treatments [9][12]
超越CRISPR:基于ADAR的RNA编辑技术,正在重塑癌症等疾病的治疗
生物世界· 2025-11-10 04:05
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]