AAV

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AAV-mediated expression of p65shRNA and bone morphogenetic protein 4 synergistically enhances chondrocyte regeneration
《中国组织工程研究》2025年第17期3537-3547,共11页Yu Yangyi Song Zhuoyue Lian Qiang Ding Kang Li Guangheng 
国家自然科学基金面上项目(81472136),项目负责人:李广恒。
BACKGROUND:Adeno-associated virus(AAV)gene therapy has been proven to be reliable and safe for the treatment of osteoarthritis in recent years.However,given the complexity of osteoarthritis pathogenesis,single gene ma...
关键词:OSTEOARTHRITIS adeno-associated virus bone morphogenetic protein 4 p65-short hairpin RNA gene therapy short hairpin RNA transforming growth factor-β1 extracellular matrix articular cartilage chondrocytes. 
基于AAV的遗传性耳聋基因治疗的基础和临床研究进展
《中国科学:生命科学》2025年第4期697-710,共14页周吟毅 杨雪寒 陆宜成 王晓涵 陈相琰 范瑾怡 张心茹 伍贤敏 张李燕 谈方志 齐洁玉 柴人杰 
国家重点研发计划(批准号:2021YFA1101300,2021YFA1101800,2020YFA0113600,2020YFA0112503);国家自然科学基金(批准号:82330033,82030029,92149304,82000984,82371162,82371161);江苏省自然科学基金基础研究计划(批准号:BK20232007);四川省科技计划重点研发项目(批准号:2021YFS0371);深圳市科技计划(批准号:JCYJ20190814093401920,JCYJ20210324125608022);广东省医学科学院2022年度开放课题(批准号:YKY-KF202201)资助。
听力障碍作为发病率极高的社会健康问题受到广泛关注.约60%的感音神经性耳聋由遗传物质突变引起.遗传性聋的临床治疗主要依赖人工耳蜗和助听器,更为有效全面的生物学治疗手段的开发迫在眉睫.修复受损的基因,是从根本上治疗遗传性耳聋的...
关键词:听力损失 基因治疗 遗传性耳聋 耳蜗 
TFE3-mediated neuroprotection:Clearance of aggregatedα-synuclein and accumulated mitochondria in the AAV-α-synuclein model of Parkinson's disease
《Genes & Diseases》2025年第2期368-382,共15页Xin He Mulan Chen Yepeng Fan Bin Wu Zhifang Dong 
supported by the National Natural Science Foundation of China (No.32371030,82071395);the CQMU Program for Youth Innovation in Future Medicine (Chongqing,China) (No.wo044);the Natural Science Foundation of Chongqing,China (No.CSTB2022NSCQBHX0022,CSTB2024NSCQ-LZX0008).
Parkinson's disease(PD)is a neurodegenerative disorder characterized by fibrillar neuronal inclusions containing aggregatedα-synuclein(α-Syn).While the pathology of PD is multi-faceted,the aggregation ofα-Syn and m...
关键词:Α-SYNUCLEIN Autophagy Mitochondrial biogenesis MITOPHAGY Parkinson's disease TFE3 
AAV载体介导的Sall2过表达延缓肌萎缩侧索硬化症转基因小鼠疾病进展
《解剖学报》2025年第2期127-135,共9页张雪 王晨晨 高学帅 白雪 王雪枚 刘金梦 管英俊 陈燕春 
国家自然科学基金(82271483);山东省自然科学基金(ZR2020MH150);山东省高等学校青创科技支持计划项目(2019KJK004)。
目的检测过表达婆罗双树样基因2(Sall2)对人超氧化物歧化酶1(hSOD1)-G93A突变型肌萎缩侧索硬化症(ALS)转基因小鼠疾病进展的影响,为ALS基因治疗提供潜在靶点。方法通过生物信息学分析筛选出差异基因Sall2。选取48只ALS转基因小鼠,通过...
关键词:肌萎缩侧索硬化症 腺相关病毒 婆罗双树样基因2 免疫印迹法 转基因小鼠 
AAV患者感染的预测列线图模型构建
《临床医学研究与实践》2025年第5期5-8,共4页周欣 李辛 冯诚怿 范紫芳 狄佳 
江苏现代医院管理研究中心2019年度课题(No.JSY-3-2019-098)。
目的构建抗中性粒细胞胞浆抗体相关性血管炎(AAV)患者感染的预测列线图模型。方法回顾性分析2010年1月至2023年6月于常州市第一人民医院就诊的193例AAV住院患者的临床资料,分别使用单因素和LASSO回归分析患者发生感染的相关因素,建立列...
关键词:抗中性粒细胞胞浆抗体相关性血管炎 感染 列线图模型 
Erratum to:Stereotactic Injection of shRNA GSK-3β-AAV Promotes Axonal Regeneration after Spinal Cord Injury
《Current Medical Science》2025年第1期154-155,共2页Yu-chao Zuo Nan-xiang Xiong Hong-yang Zhao 
Erratum to:J Huazhong Univ Sci Technol[Med Sci]36(4):548–553,2016 https://doi.org/10.1007/s11596-016-1623-6 In the originally published article(https://doi.org/10.1007/s11596-016-1623-6),the immunofluorescence images...
关键词:spinal cord injury stereotactic injection GSK axonal regeneration immunofluorescence images SHRNA AAV shrna group 
Identification of AAV serotypes for gene therapy in Krabbe iPSCs-derived brain organoids
《Genes & Diseases》2025年第1期144-147,共4页Yafeng Lv Zhi Cui Hongbo Li Jing Wang Mulan Wei Yuanlang Hu Xun Li Chunyu Cao Ye Zhang Wei Wang 
funded by grants from the Beijing Municipal Science&TechnologyCommission(China)(No.Z211100002921005 to W.W.);National High-Level Hospital Clinical Research Funding(China)(No.2022-NHLHCRF-PY-12 to W.W.);Hubei Provincial Department of Education's Scientific and Technological Research Project(China)(No.Q20211207 to Y.L.);Yichang Medical and Health Science and Technology Project(Hubei,China)(No.A22-2-069 to Y.L.);the Open Foundation of Hubei Province Key Laboratory of Tumor Microenvironment and Immunotherapy(China)(No.2023KZL08 to Y.L.).
Krabbe disease,also known as globoid cell leukodystrophy,is a rare lysosomal storage disorder.It is primarily caused by mutations in the GALC gene on chromosome 14q31,leading to GALC enzyme deficiency in lysosomes.Thi...
关键词:AAV DYSTROPHY diseases 
Current AAV-mediated gene therapy in sensorineural hearing loss
《Fundamental Research》2025年第1期192-202,共11页Jieyu Qi Xiaolong Fu Liyan Zhang Fangzhi Tan Nianci Li Qiuhan Sun Xiaojie Hu Zuhong He Ming Xia Renjie Chai 
supported by the National Key Research and Development Program of China(2020YFA0113600,2021YFA1101300,2020YFA0112503);Strategic Priority Research Program of the Chinese Academy of Science(XDA16010303);the National Natural Science Foundation of China(82000984,82030029,81970882,81900937 and 82071013);Natural Science Foundation from Jiangsu Province(BE2019711);the China National Postdoctoral Program for Innovative Talents(BX20200082);the China Postdoctoral Science Foundation(2020M681468);the Science and Technology Department of Sichuan Province(2021YFS0371);Shenzhen Fundamental Research Program(JCYJ20190814093401920,JCYJ20210324125608022);Open Research Fund of State Key Laboratory of Genetic Engineering,Fudan University(SKLGE-2109);the Fundamental Research Funds for the Central Universities.
The number of patients with hearing loss is on the rise due to congenital abnormalities,degenerative changes in old age,and acquired injuries such as virus or ototoxic drug-induced diseases.Hearing loss is a refractor...
关键词:Hearing loss COCHLEA Gene therapy Adeno-associated virus Auditory repair 
AAV基因治疗遗传性挛性截瘫50型患者的临床研究
《中国临床药理学杂志》2025年第3期427-427,共1页
个性化基因治疗是治疗这些疾病的一种有希望的方法。该研究通过为一位患有遗传性痉挛性截瘫50型(spastic paraplegia type 50,SPG50)的患者开发基因替代疗法,提出了个性化治疗超罕见疾病的路线图。通过多中心合作,该研究构建了1种携带...
关键词:SPG50 个性化基因治疗 遗传性痉挛性截瘫 
AAV mediated carboxyl terminus of Hsp70 interacting protein overexpression mitigates the cognitive and pathological phenotypes of APP/PS1 mice
《Neural Regeneration Research》2025年第1期253-264,共12页Zhengwei Hu Jing Yang Shuo Zhang Mengjie Li Chunyan Zuo Chengyuan Mao Zhongxian Zhang Mibo Tang Changhe Shi Yuming Xu 
supported by the National Natural Science Foundation of China,Nos.91849115 and U1904207(to YX),81974211 and 82171247(to CS);Non-profit Central Research Institute Fund of Chinese Academy of Medical Sciences,No.2020-PT310-01(to YX).
The E3 ubiquitin ligase,carboxyl terminus of heat shock protein 70(Hsp70)interacting protein(CHIP),also functions as a co-chaperone and plays a crucial role in the protein quality control system.In this study,we aimed...
关键词:adeno-associated virus Alzheimer’s disease APP/PS1 mice carboxyl terminus of Hsp70 interacting protein gene therapy 
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