机构：[1]Zhongmou Theraput, Wuhan, Hubei, Peoples R China[2]Beijing Tongren Eye Ctr, Beijing, Peoples R China首都医科大学附属北京同仁医院首都医科大学附属同仁医院[3]Beijing Tongren Hosp, Beijing, Peoples R China首都医科大学附属北京同仁医院首都医科大学附属同仁医院[4]Wuahan Univ, Wuhan, Hubei, Peoples R China
出处：
ISSN：
摘要：
Purpose : Retinitis pigmentosa (RP) is the most common inherited retinal disease, affecting over 200 million people globally, and it has no approved treatment. Advanced RP causes significant decreases in visual acuity, visual field, and quality of life. ZM-02 is an optogenetic gene therapy that uses an adeno-associated virus to deliver a novel photosensitive protein gene, PsCatCh2.0, via intravitreal injection. Thus, we conducted a MOON trial (NCT06292650) to evaluate ZM-02 in advanced RP patients with various causative genes.
基金：
National Key R&D Program of China 2017YFE0103400; National Nature Science Foundation of China 81800872; National Nature Science Foundation of China 81900874 and 82104490; the Fundamental Research Funds for the Central Universities 2042022dx0003
语种：
外文
WOS：
中科院(CAS)分区：
出版当年[2025]版：
大类|1 区医学
小类|1 区生物工程与应用微生物1 区遗传学1 区医学：研究与实验
最新[2025]版：
大类|1 区医学
小类|1 区生物工程与应用微生物1 区遗传学1 区医学：研究与实验
JCR分区：
出版当年[2023]版：
Q1BIOTECHNOLOGY & APPLIED MICROBIOLOGYQ1GENETICS & HEREDITYQ1MEDICINE, RESEARCH & EXPERIMENTAL
最新[2024]版：
Q1BIOTECHNOLOGY & APPLIED MICROBIOLOGYQ1GENETICS & HEREDITYQ1MEDICINE, RESEARCH & EXPERIMENTAL
第一作者机构：[4]Wuahan Univ, Wuhan, Hubei, Peoples R China
推荐引用方式(GB/T 7714)：
Zhou Wenhui,Yin Shiyi,Zhang Haihan,et al.Safety and Efficacy of ZM-02 Optogenetic Gene Therapy Clinical Study for Advanced Retinitis Pigmentosa[J].MOLECULAR THERAPY.2025,33(4):
APA：
Zhou, Wenhui,Yin, Shiyi,Zhang, Haihan,Zhou, Menghan,Shirinzadeh, Mojtaba...&Wei, Wenbin.(2025).Safety and Efficacy of ZM-02 Optogenetic Gene Therapy Clinical Study for Advanced Retinitis Pigmentosa.MOLECULAR THERAPY,33,(4)
MLA：
Zhou, Wenhui,et al."Safety and Efficacy of ZM-02 Optogenetic Gene Therapy Clinical Study for Advanced Retinitis Pigmentosa".MOLECULAR THERAPY 33..4(2025)
医学