GENE_THERAPY

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Long-term correction of hemorrhagic diathesis in hemophilia A mice by an AAV-delivered hybrid FⅧcomposed of the human heavy chain and the rat light chain
《Frontiers of Medicine》2022年第4期584-595,共12页Jianhua Mao Yun Wang Wei Zhang Yan Shen Guowei Zhang Wenda Xi Qiang Wang Zheng Ruan Jin Wang Xiaodong Xi 
supported by the National Key Basic Research Program of China(No.2013CB966800);National Natural Science Foundation of China(Nos.81970112,81670127,and 81101721);the Novo Nordisk Hemophilia Foundation,grants from the Shanghai Health Commission in China(No.201940342);grants from the Science and Technology Commission of Shanghai Municipality in China(Nos.16PJ1406100 and 16ZR1421000);Zhejiang Provincial Natural Science Foundation of China(No.LY17H080004).
Conventional therapies for hemophilia A(HA)are prophylactic or on-demand intravenous FⅧinfusions.However,they are expensive and inconvenient to perform.Thus,better strategies for HA treatment must be developed.In thi...
关键词：hemophilia A adeno-associated virus(AAV) human/rat hybrid factorⅧ gene therapy dual chain strategy 
Dual-targeted lung cancer therapy via inhalation delivery of UCNP-siRNA-AS1411 nanocages
《Cancer Biology & Medicine》2022年第7期1047-1060,共14页Yu Han Yuming Yang Qiuyang Sun Bin Li Caixia Yue Yanlei Liu Jesús M.de la Fuente Daxiang Cui 
supported by the National Key Basic Research Program(973 Project)(Grant Nos.2015CB931802 and 2017FYA0205301);the Special Fund for Science and Technology Innovation of Shanghai Jiao Tong University(Grant Nos.YG2017MS70,YG2015MS62,and AF0300179);the Shanghai Municipal Bureau of Economy and Information Technology(Grant No.XC-ZXSJ-02-2016-05);the National Natural Scientific Foundation of China(Grant Nos.8202010801,81921002,81225010,81028009,and 31170961);the 863 Project of China(Grant No.2014AA020700);and the Shanghai Science and Technology Fund(Grant No.13NM1401500)。
Objective:Although great progress has been made in the field of siRNA gene therapy,safe,efficient,and targeted delivery of siRNA are still major challenges in siRNA therapeutics.Methods:We developed an up-conversion n...
关键词：Nanomaterials VEGF siRNA lung cancer gene therapy siRNA delivery 
AAV-ie-K558R mediated cochlear gene therapy and hair cell regeneration被引量：4
《Signal Transduction and Targeted Therapy》2022年第5期1764-1772,共9页Yong Tao Xiaoyi Liu Liu Yang Cenfeng Chu Fangzhi Tan Zehua Yu Junzi Ke Xiang Li Xiaofei Zheng Xingle Zhao Jieyu Qi Chao-Po Lin Renjie Chai Guisheng Zhong Hao Wu 
We thank the Shanghai Municipal Government and ShanghaiTech University,and the Bioimaging Core Facilities of the iHuman Institute and the animal facility of National Center for Protein Science Shanghai for their financial support.Hao Wu was supported by the Key Project of the National Natural Science Foundation of China(NSFC 81730028);the National Key Technology Research and Development Program of the Ministry of Science and Technology of China(2017YFC1001800);Shanghai Key Laboratory of Translational Medicine on Ear and Nose diseases(14DZ2260300);Shanghai Municipal Science and Technology Major Project(2018SHZDZX05);the Innovative Research Team of High-level Local Universities in Shanghai.This work was supported by the National Natural Science Foundation of China(31771130(G.Z.);the 2015 Thousand Youth Talents Plan of China(G.Z.,).This work was supported by the National Natural Science Foundation of China(82122019,Y.T.);the program for professors with special appointments(Eastern Scholar,Y.T.)at Shanghai Institutions of Higher Learning.
The cochlea consists of multiple types of cells,including hair cells,supporting cells and spiral ganglion neurons,and is responsible for converting mechanical forces into electric signals that enable hearing.Genetic a...
关键词：COCHLEAR AUDITORY DEAFNESS 
Gene therapy for neurodegenerative disorders:advances, insights and prospects被引量：10
《Acta Pharmaceutica Sinica B》2020年第8期1347-1359,共13页Wei Chen Yang Hu Dianwen Ju 
supported by National Natural Science Foundation of China(Nos.81773620 and 81573332);National Key Basic Research Program of China(No.2015CB931800)to Dianwen Ju;NIH NEI(EY024932,EY023295 and EY028106,USA)to Yang Hu
Gene therapy is rapidly emerging as a powerful therapeutic strategy for a wide range of neurodegenerative disorders, including Alzheimer’s disease(AD), Parkinson’s disease(PD) and Huntington’s disease(HD). Some ear...
关键词：DISORDERS DEGENERATIVE HUNTINGTON 
A review of application of base editing for the treatment of inner ear disorders
《Journal of Bio-X Research》2020年第2期66-71,共6页Zhao Xingle Sun Zhuoer Kang Wen Tao Yong Wu Hao 
HW was supported by the Key Project of National Natural Science Foundation of China(No.81330023);the National Key Technology Research and Development Program of the Ministry of Science and Technology of China(No.SQ2017YFSF080012);Shanghai Key Laboratory of Translational Medicine on Ear and Nose diseases,China(Nos.14DZ2260300);Innovative Research Team of High-level Local Universities in Shanghai,China.YT was supported by National Natural Science Foundation of China(No.NSFC81800900);Shanghai Science and Technology Committee,China(Nos.18411953600,18ZR1422100,18PJ1406900);Shanghai Municipal Health Commission,China(No.2018YQ59);Shanghai Ninth People’s Hospital,China(No.QC201804).
The development of applications for the CRISPR/Cas9(clustered regularly interspaced short palindromic repeats/CRISPR-associated nuclease 9)system has increased greatly in recent years,especially in the area of gene th...
关键词：base editing base editor CRISPR deafness treatment gene editing gene therapy HEARING inner ear 
Multifunctional nucleic acid nanostructures for gene therapies被引量：4
《Nano Research》2018年第10期5017-5027,共11页Jianbing Liu Zhengang Wang Shuai Zhao Baoquan Ding 
This work is supported by the National Natural Science Foundation of China （Nos. 21573051, 21708004, and 51761145044）, Sience Fund of Creative Research Groups of the National Natural Science Foundation of China （No. 21721002）, the National Basic Research Program of China （No. 2016YFA0201601）, Beijing Municipal Science ＆ Technology Commission （No. Z161100000116036）, Key Research Program of Frontier Sciences, CAS, Grant QYZDB-SSW-SLH029, CAS Interdisciplinary Innovation Team, and K. C. Wong Education Foundation.
Nucleic acid nanotechnology has been developed to be a promising strategy to construct various nano-biomaterials with structural programmability, spatial addressability, and excellent biocompatibility. Self-assembled ...
关键词：nucleic acid nanostructure nucleic acid drug gene therapy drug delivery multifunctional nanomaterials 
Adenovirus-mediated gene delivery:Potential applications for gene and cell-based therapies in the new era of personalized medicine被引量：24
《Genes & Diseases》2017年第2期43-63,共21页Cody S.Lee Elliot S.Bishop Ruyi Zhang Xinyi Yu Evan M.Farina Shujuan Yan Chen Zhao Zongyue Zeng Yi Shu Xingye Wu Jiayan Lei Yasha Li Wenwen Zhang Chao Yang Ke Wu Ying Wu Sherwin Ho Aravind Athiviraham Michael J.Lee Jennifer Moriatis Wolf Russell R.Reid Tong-Chuan He 
Research in the authors’laboratories was supported in part by research grants from the National Institutes of Health(AT004418,DE020140 to TCH and RRR);the US Department of Defense(OR130096 to JMW);the Scoliosis Research Society(TCH and MJL);the 973 Program of the Ministry of Science and Technology(MOST)of China(#2011CB707906 to TCH);The reported work was also supported in part by The University of Chicago Cancer Center Support Grant(P30CA014599);the National Center for Advancing Translational Sciences of the National Institutes of Health through Grant Number UL1 TR000430.
With rapid advances in understanding molecular pathogenesis of human diseases in the era of genome sciences and systems biology,it is anticipated that increasing numbers of therapeutic genes or targets will become ava...
关键词：ADENOVIRUS Adenoviral vector Cell therapy Gene transfer Gene therapy Oncolytic virus Regenerative medicine Vaccine development 
Genome Editing and Its Applications in Model Organisms被引量：3
《Genomics, Proteomics & Bioinformatics》2015年第6期336-344,共9页Dongyuan Ma Feng Liu 
supported by grants from the National Basic Research Program of China (Grant Nos. 2010CB945300 and 2011CB943900);the National Natural Science Foundation of China (Grant Nos. 31271570, 31425016, 31370030, and 81530004);the Strategic Priority Research Program of the Chinese Academy of Sciences (Grant No. XDA01010110)
Technological advances are important for innovative biological research. Development of molecular tools for DNA manipulation, such as zinc finger nucleases （ZFNs）, transcription activator-like effector nucleases （T...
关键词：Genome editingCRISPR/Cas ZEBRAFISH Disease model Gene therapy 
Optimized human factor IX expression cassettes for hepaticdirected gene therapy of hemophilia B被引量：2
《Frontiers of Medicine》2015年第1期90-99,共10页Ru Zhang Qiang Wang Lin Zhang Saijuan Chen 
Gene therapy provides a potential cure for hemophilia B, and significant progress has been achieved in liver-directed gene transfer mediated by adeno-associated viral vectors. Recent clinical trials involving the use ...
关键词：factor IX hemophilia B liver-specific regulatory elements hydrodynamic gene transfer 
Correction of a genetic disease by CRISPR-Cas9-mediated gene editing in mouse spermatogonial stem cells被引量：41
《Cell Research》2015年第1期67-79,共13页Yuxuan Wu Hai Zhou Xiaoying Fan Ying Zhang Man Zhang Yinghua Wang Zhenfei Xie Meizhu Bai Qi Yin Dan Liang Wei Tang Jiaoyang Liao Chikai Zhou Wujuan Liu Ping Zhu Hongshan Guo Hong Pan Chunlian Wu Huijuan Shi Ligang Wu Fuchou Tang Jinsong Li 
This study was supported by the Ministry of Science and Technology of China （2014CB964803 and 2011CB811304 to JL, 2012CB966704 and 2011CB66303 to FT, 2014CB943104 to LW）, the National Natural Science Foundation of China （31225017 and 91319310 to JL, 31271543 and 31322037 to FT）, the Strategic Priority Research Program of the Chinese Academy of Sciences （XDA01010403）, and the Shanghai Municipal Commission for Science and Technology （12JC1409600 and 13XD1404000 to JL, 12JC 1409400 to LW）.
Spermatogonial stem cells （SSCs） can produce numerous male gametes after transplantation into recipient testes, presenting a valuable approach for gene therapy and continuous production of gene-modified animals. How...
关键词：CRISPR-Cas9 spermatogonial stem cell gene therapy 
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