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Gene replacement therapy in Bietti crystalline corneoretinal dystrophy:an open-label,single-arm,exploratory trial

Signal Transduction and Targeted Therapy 2024年第5期9卷 2275-2284页

作者： Jinyuan Wang Jinlu Zhang shicheng yu Hongyan Li Shaohong Chen Jingting Luo Haibo Wang yuxia Guan Haihan Zhang Shiyi Yin Huili Wang Heping Li Junle Liu Jingyuan Zhu Qiong Yang Ying Sha Chuan Zhang yuhang Yang Xuan Yang Xifang Zhang Xiuli Zhao Likun Wang Liping Yang Wenbin Wei Beijing Tongren Eye Center Beijing Key Laboratory of Intraocular Tumor Diagnosis and TreatmentBeijing Ophthalmology&Visual Sciences Key LabMedical Artificial Intelligence Research and Verification Key Laboratory of the Ministry of Industry and Information TechnologyBeijing Tongren HospitalCapital Medical University100730 BeijingChina School of Clinical Medicine Tsinghua University100084 BeijingChina Chigenovo Co. Ltd.102206 BeijingChina Department of Ophthalmology Peking University Third HospitalBeijing Key Laboratory of Restoration of Damaged Ocular NervePeking University Third Hospital100191 BeijingChina Department of Ophthalmology The Third People’s Hospital of Dalian116091 DalianChina Department of Biomedical Informatics School of Basic Medical SciencesPeking University Health Science Center100191 BeijingChina Institute of Systems Biomedicine Department of PathologySchool of Basic Medical SciencesPeking University Health Science Center100191 BeijingChina

Bietti crystalline corneoretinal dystrophy is an inherited retinal disease caused by mutations in CYP4V2,which results in blindness in the working-age population,and there is currently no available treatment.Here,we report the results of the first-in-human clinical trial(NCT04722107)of gene therapy for Bietti crystalline corneoretinal dystrophy,including 12 participants who were followed up for 180-365 days.This open-label,single-arm exploratory trial aimed to assess the safety and efficacy of a recombinant adeno-associated-virus-serotype-2/8 vector encoding the human CYP4V2 protein(rAAV2/8-hCYP4V2).Participants received a single unilateral subretinal injection of 7.5×10^(10)vector genomes of rAAV2/8-hCYP4V2.Overall,73 treatment-emergent adverse events were reported,with the majority(98.6%)being of mild or moderate intensity and considered to be procedure-or corticosteroid-related;no treatment-related serious adverse events or local/systemic immune toxicities were observed.Compared with that measured at baseline,77.8%of the treated eyes showed improvement in best-corrected visual acuity(BCVA)on day 180,with a mean±standard deviation increase of 9.0±10.8 letters in the 9 eyes analyzed(p=0.021).By day 365,80%of the treated eyes showed an increase in BCVA,with a mean increase of 11.0±10.6 letters in the 5 eyes assessed(p=0.125).Importantly,the patients’improvement observed using multifocal electroretinogram,microperimetry,and Visual Function Questionnaire-25 further supported the beneficial effects of the treatment.We conclude that the favorable safety profile and visual improvements identified in this trial encourage the continued development of rAAV2/8-hCYP4V2(named ZVS101e).

关键词： cor dystrophy crystalline

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Sequential reactant water management by complementary multisite catalysts for surpassing platinum hydrogen evolution activity

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Nano Research 2024年第3期17卷 1232-1241页

作者： yu Lin Defang Ding shicheng Zhu Qunlei Wen Huangjingwei Li Zhen Li Youwen Liu Yi Shen China Engineering Research Center of Nano-Geomaterials of Ministry of Education Faculty of Materials Science and ChemistryChina University of GeosciencesWuhan 430078China State Key Laboratory of Materials Processing and Die&Mould Technology and School of Materials Science and EngineeringHuazhong University of Science and TechnologyWuhan 430074China

Alkaline hydrogen evolution reaction(HER)offers a near-zero-emission approach to advance hydrogen energy.However,the activity limited by the multiple reaction steps involving H_(2)O molecules transfer,absorption,and activation still unqualified the thresholds of economic viability.Herein,we proposed a multisite complementary strategy that incorporates hydrophilic Mo and electrophilic V into Ni-based catalysts to divide the distinct steps on atomically dispersive sites and thus realize sequential regulation of the HER process.The Isotopic labeled in situ Raman spectroscopy describes 4-coordinated hydrogen bonded H_(2)O to be free H_(2)O passing the inner Helmholtz plane in the vicinity of the catalysts under the action of hydrophilic Mo sites.Furthermore,potential-dependent electrochemical impedance spectroscopy(EIS)reveals that electrophilic V sites with abundant 3d empty orbitals could activate the lone-pair electrons in the free H_(2)O molecules to produce more protic hydrogen,and dimerize into H_(2) at the Ni sites.By the sequential management of reactive H_(2)O molecules,NiMoV oxides multisite catalysts surpass Pt/C hydrogen evolution activity(49 mV@10 mA∙cm^(-2) over 140 h).Profoundly,this study provides a tangible model to deepen the comprehension of the catalyst–electrolyte interface and create efficient catalysts for diverse reactions.

关键词： hydrogen evolution activity complementary multisite catalysts sequential reactive water management interfacial water molecules

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Comparing COVID-19 Case Prediction Between ARIMA Model and Compartment Model—China,December 2019–April 2020

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China CDC weekly 2022年第52期4卷 1185-1188,I0006-I0010页

作者： Bangguo Qi Nankun Liu shicheng yu Feng Tan Chinese Center for Disease Control and Prevention BeijingChina.

Introduction:To compare the performance between the compartment model and the autoregressive integrated moving average(ARIMA)model that were applied to the prediction of new infections during the coronavirus disease 2019(COVID-19)epidemic.Methods:The compartment model and the ARIMA model were established based on the daily cases of new infection reported in China from December 2,2019 to April 8,2020.The goodness of fit of the two models was compared using the coefficient of determination(R2).Results:The compartment model predicts that the number of new cases without a cordon sanitaire,i.e.,a restriction of mobility to prevent spread of disease,will increase exponentially over 10 days starting from January 23,2020,while the ARIMA model shows a linear increase.The calculated R2 of the two models without cordon sanitaire were 0.990 and 0.981.The prediction results of the ARIMA model after February 2,2020 have a large deviation.The R2 of complete transmission process fit of the epidemic for the 2 models were 0.964 and 0.933,respectively.Discussion:The two models fit well at different stages of the epidemic.The predictions of compartment model were more in line with highly contagious transmission characteristics of COVID-19.The accuracy of recent historical data had a large impact on the predictions of the ARIMA model as compared to those of the compartment model.&Joint first authors.

关键词： ARIMA prediction starting

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The Health Demands of Designated Drivers-Four Cities,China,2019

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China CDC weekly 2022年第20期4卷 426-430页

作者： Meng Zhang Qiqi Wang Hongyan Yao Xiao Qi shicheng yu Jianjun Liu Office of Epidemiology Chinese Center for Disease Control and PreventionBeijingChina Chinese Center for Disease Control and Prevention BeijingChina

Summary What is already known about this topic?The health status of designated drivers is largely associated with road safety,which is a major public health issue.However,few studies have focused on the health demands of designated drivers.What is added by this report?This study investigated the health consciousness,first aid knowledge learning,acceptable ways to acquire health knowledge,and willingness to have physical examinations for designated drivers to provide suggestions for improving their health status.What are the implications for public health practice?The industry and platform should provide scientific and reasonable guidance on healthy lifestyles for designated drivers and implement physical examinations to monitor their health status.

关键词： designated knowledge consciousness

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Application of Disability-Adjusted Life Years to Evaluate the Burden and Trend of Tuberculosis — China, 1990–2019

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China CDC weekly 2022年第11期4卷 220-225页

作者： Zhe Dong Qiqi Wang shicheng yu Jianjun Liu Hui Zhang Fei Huang Hongyan Yao Chinese Center for Disease Control and Prevention BeijingChina

Summary What is already known about this topic?China is a country with a high burden of tuberculosis(TB).However,the evaluation of TB burden is usually based on traditional epidemiological indicators,and disability-adjusted life year(DALY)is rarely applied.What is added by this report?In China,the number of DALYs caused by TB in 2019 was estimated to be 1.36 million,and the agestandardized rate(ASR)was 86.46/100,000.The DALYs of TB decreased significantly from 1990 to 2019,with the largest decline from 2001 to 2010.The burden is higher among men than women,and the highest among the elderly.What are the implications for public health practice?Application of DALYs can reflect the damage in health to China’s residents caused by tuberculosis.In the future,we should strengthen research,monitor years lived with disability(YLDs),and reduce the burden of the elderly and men.Cost-effective analysis based on DALYs should be conducted to provide a scientific basis for decision-making.

关键词： Application burden elderly

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Intestinal cellular heterogeneity and disease development revealed by single-cell technology

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Cell Regeneration 2022年第1期11卷 267-289页

作者： Yalong Wang Wanlu Song shicheng yu yuan Liu Ye-Guang Chen Guangzhou Institutes of Biomedicine and Health Chinese Academy of Sci-encesGuangzhou Science ParkLuogang DistrictGuangzhouChina Guang-zhou Laboratory GuangzhouChina The State Key Laboratory of Membrane Biology Tsinghua-Peking Center for Life SciencesSchool of Life SciencesTsinghua UniversityBeijingChina

The intestinal epithelium is responsible for food digestion and nutrient absorption and plays a critical role in hormone secretion,microorganism defense,and immune response.These functions depend on the integral single-layered intestinal epithelium,which shows diversified cell constitution and rapid self-renewal and presents powerful regen-eration plasticity after injury.Derailment of homeostasis of the intestine epithelium leads to the development of dis-eases,most commonly including enteritis and colorectal cancer.Therefore,it is important to understand the cellular characterization of the intestinal epithelium at the molecular level and the mechanisms underlying its homeostatic maintenance.Single-cell technologies allow us to gain molecular insights at the single-cell level.In this review,we summarize the single-cell RNA sequencing applications to understand intestinal cell characteristics,spatiotemporal evolution,and intestinal disease development.

关键词： scRNA-seq Gut Heterogeneity Enteritis Colorectal cancer

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Economic Burden of Malignant Tumors——Yichang City,Hubei Province,China,2019

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China CDC weekly 2022年第15期4卷 312-316页

作者： Xiaojuan Long Fangfang Lu Xianglong Xiang Jiajuan Yang Chengzhong Xu Pei Zhang shicheng yu Qiqi Wang Chi Hu Office of Epidemiology Chinese Center for Disease Control and PreventionBeijingChina Yichang Center for Disease Control and Prevention Yichang CityHubei ProvinceChina

Summary What is already known about this topic?Malignant tumors are common chronic noncommunicable disease and have caused serious health hazards to residents and heavy economic burden of disease to the society.What is added by this report?This is the first report on the economic burden of multiple types of malignant tumors in Yichang City.In 2019,the direct medical burden of lung cancer in Yichang was the highest,reaching 561.67 million CNY,and the indirect economic burden of lung cancer in Yichang was higher than that of other malignant tumors,costing 326.49 million CNY.

关键词： Malignant lung burden

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Professor Jian Lu’s Interdisciplinary Approach to Modeling the COVID-19 Pandemic

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China CDC weekly 2020年第27期2卷 511-513页

作者： Peter Hao shicheng yu yu Chen Feng Tan Chinese Center for Disease Control and Prevention BeijingChina National Institute of Environmental Health Chinese Center for Disease Control and PreventionBeijingChina

Editorial Our new column Insights is an ongoing series of interviews prepared by the China CDC Weekly team with researchers,policymakers,experts,and other leaders that strives to disseminate experiences,knowledge,and perspectives in global and public health with a wider community.At the discretion of the Weekly’s team,these interviews will be written into articles,shared as videos,and/or presented as transcripts to best promote plain-language principles and to ease understanding of non-specialized academic readers.

关键词： dis ongoing specialized

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积分型非奇异终端滑模PMSM无传感器控制系统

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电机与控制学报 2024年第3期28卷 169-178页

作者：郑诗程刘志鹏赵卫王宇郎佳红安徽工业大学电气与信息工程学院安徽马鞍山243000

针对永磁同步电机中高速情况下传统的滑模观测器估算精度低且存在较强抖振的问题,提出一种基于改进型滑模观测器的PMSM矢量控制方法。基于非线性滑模面理论分析,构建一种积分型非奇异终端滑模面,有效降低了抖振现象,提高了系统的观测精... 详细信息

针对永磁同步电机中高速情况下传统的滑模观测器估算精度低且存在较强抖振的问题,提出一种基于改进型滑模观测器的PMSM矢量控制方法。基于非线性滑模面理论分析,构建一种积分型非奇异终端滑模面,有效降低了抖振现象,提高了系统的观测精确度;并设计了一种自适应反电动势滤波器,使反电动势能随观测器自适应调节,且谐波含量低,进一步提升动态精度;最后,利用正交锁相环原理调制出电机转子位置信息,将提出的新型控制方法应用到永磁同步电机调速系统,与传统滑模控制进行对比。仿真和实验表明,提出的基于新型滑模观测器的永磁同步电机控制系统跟踪精度高、鲁棒性强,动、静态响应好。

关键词：反电动势滤波器永磁同步电机无传感器控制滑模观测器调速系统

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Incidence Tendency Analysis on Type 2 Diabetes in 4 Asian Countries—China,Malaysia,Singapore,and Thailand,1990–2019

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China CDC weekly 2021年第52期3卷 1113-1117页

作者： Wenhui Shi Qiqi Wang Jiaojiao Zhang Jingwen Zhang Feng Tan shicheng yu Division of Non-communicable Diseases and Elderly Health Management Chinese Center for Disease Control and PreventionBeijingChina Office of Epidemiology Chinese Center for Disease Control and PreventionBeijingChina Institute of Infectious Diseases Beijing Center for Disease Control and PreventionBeijingChina Shijiazhuang Finance Bureau of Hebei Province ShijiazhuangHebeiChina Division of Academic Publishing Management Chinese Center for Disease Control and PreventionBeijingChina.

Summary What is already known about this topic?Roughly 80%of the global disease burden caused by diabetes comes from low-and-middle income countries,and 60%of diabetics are located in Asia;6 of the top 10 countries with the highest prevalence of diabetes are in Asia.What is added by this report?Compared with 1990,the growth rate of the standardized incidence rate of type 2 diabetes in 2019 in China was significantly lower than that of the entire world,while Malaysia,Singapore.

关键词： Diabetes income Singapore

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