ADENO-ASSOCIATED

作品数：12被引量：40H指数：3
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相关领域：医药卫生生物学更多>>
相关作者：娄智勇贾书勤更多>>
相关机构：首都医科大学清华大学更多>>
相关期刊：《World Journal of Translational Medicine》《Science China(Life Sciences)》《World Journal of Gastroenterology》《Science Bulletin》更多>>
相关基金：国家自然科学基金国家高技术研究发展计划更多>>
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Enhanced genome editing to ameliorate a genetic metabolic liver disease through co-delivery of adeno-associated virus receptor被引量：2
《Science China(Life Sciences)》2022年第4期718-730,共13页Shuming Yin Lie Ma Tingting Shao Mei Zhang Yuting Guan Liren Wang Yaqiang Hu Xi Chen Honghui Han Nan Shen Wenjuan Qiu Hongquan Geng Yongguo Yu Shichang Li Weishi Yu Mingyao Liu Dali Li 
partially supported by grants from the National Key R&D Program of China (2019YFA0110802);the National Natural Science Foundation of China (81670470 and 81873685);grants from the Shanghai Municipal Commission for Science and Technology (18411953500 and 20140900201);a grant from the Innovation Program of Shanghai Municipal Education Commission (2019-01-07-00-05-E00054);the Fundamental Research Funds for the Central Universities
Genome editing through adeno-associated viral(AAV) vectors is a promising gene therapy strategy for various diseases,especially genetic disorders. However, homologous recombination(HR) efficiency is extremely low in a...
关键词：CRISPR/Cas9 gene therapy adeno-associated virus(AAV) AAVR PAH PKU 
Characteristics and advantages of adeno-associated virus vector-mediated gene therapy for neurodegenerative diseases被引量：6
《Neural Regeneration Research》2019年第6期931-938,共8页Yuan Qu Yi Liu Ahmed Fayyaz Noor Johnathan Tran Rui Li 
Common neurodegenerative diseases of the central nervous system are characterized by progressive damage to the function of neurons, even leading to the permanent loss of function. Gene therapy via gene replacement or ...
关键词：nerve REGENERATION central nervous system gene therapy NEURODEGENERATIVE DISEASE viral vector ADENO-ASSOCIATED virus Alzheimer’s DISEASE Parkinson’s DISEASE Huntington’s DISEASE amyotrophic lateral SCLEROSIS spinal muscular atrophy neural REGENERATION 
S6B-4 Roles of the Basal Ganglia in Sleep-Wake Regulation
《神经药理学报》2018年第4期97-98,共2页HUANG Zhi-li 
The basal ganglia(BG)act as a cohesive functional unit that regulates motor function,habit formation,and reward/addictive behaviors.However,it is still not well understood how the BG maintains wakefulness and suppress...
关键词：ADENO-ASSOCIATED virus OPTOGENETICS DREADD BASAL GANGLIA SLEEP-WAKE REGULATION 
Soluble interleukin 23 receptor gene therapy with adeno-associated vectors for the treatment of multiple sclerosis
《Neural Regeneration Research》2017年第10期1605-1606,共2页ángel Edo Juan Francisco Espinosa-Parrilla 
Gene therapy as strategy against autoimmune diseases： In the last century, human societies have witnessed remarkable improvements in wellness and life expectancy, thanks to the consolidation of mod- ern science and m...
Gene editing for corneal disease management
《World Journal of Translational Medicine》2016年第1期1-13,共13页Sudhanshu P Raikwar Apoorva S Raikwar Shyam S Chaurasia Rajiv R Mohan 
Veteran Health Affairs Merit grant,No.1I01BX000357-05 (to Mohan RR);National Eye Institute,NIH grant,R01EY017294 (to Mohan RR);the Ruth M.Kraeuchi Missouri Endowment of Ophthalmology (to Mohan RR)
Gene editing has recently emerged as a promising technology to engineer genetic modifications precisely in the genome to achieve long-term relief from corneal disorders.Recent advances in the molecular biology leading...
关键词：ADENO-ASSOCIATED virus Clustered Regularly-Interspaced SHORT Palindromic Repeats associated protein 9 Cornea Clustered regularly interspaced SHORT palindromic repeat Double strand breaks GENE EDITING sgRNA GENE targeting Homology directed repair Homologous recombination Indels LENTIVIRAL vector Protospacer-adjacent motif Transcription activator like effector NUCLEASES Zinc finger NUCLEASES 
Targeting adeno-associated virus and adenoviral genetherapy for hepatocellular carcinoma被引量：2
《World Journal of Gastroenterology》2016年第1期326-337,共12页Yi-Gang Wang Pan-Pan Huang Rong Zhang Bu-Yun Ma Xiu-Mei Zhou Yan-Fang Sun 
Supported by National Natural Science Foundation of China.No.81272687;Zhejiang Provincial Public Welfare Technology Application Research Projects,No.2014C33275;Zhejiang Provincial Natural Science Foundation of China,No.LZ13H160004;the Grant for 521 Talent Project of Zhejiang Sci-Tech University,Hangzhou,China
Human hepatocellular carcinoma(HCC)heavily endangers human heath worldwide.HCC is one of most frequent cancers in China because patients with liver disease,such as chronic hepatitis,have the highest cancer susceptibil...
关键词：Hepatocellular carcinoma Adeno-associatedvirus ADENOVIRUS VIRUS VECTORS 
Adeno-associated virus vectors for human gene therapy
《World Journal of Medical Genetics》2015年第3期28-45,共18页Haifeng Chen 
Adeno-associated virus(AAV) is a small,non-enveloped virus that contains a single-stranded DNA genome. It was the first gene therapy drug approved in the Western world in November 2012 to treat patients with lipoprote...
关键词：Adeno-associated virus Adeno-associated virus production and purification Clinical trials Gene therapy BACULOVIRUS 
Constructing and packaging scAAV vectors and detecting their transduction efficiency
《Journal of Nanjing Medical University》2007年第6期401-401,共1页Jianqing Wu Weihong Zhao 
This study was supported by the Natural Science Foundation of Jiangsu Province(BK2006244);the Key Medical Person Foundation of Jiangsu Province (RC2007051)
Gene therapy is a form of molecular medicine that promises to provide new treatments for a large number of inherited and acquired diseases. One of the biggest stumbling blocks to successful widespread application of g...
关键词：gene therapy self-complementary adeno-associated viral transduction efficiency 
A novel and highly efficient production system for recombinant adeno-associated virus vector被引量：10
《Science China(Life Sciences)》2002年第1期96-104,共9页伍志坚 吴小兵 曹晖 董小岩 王宏 侯云德 
This work was supported by the National High Technology Development Program.
Recombinant adeno-associated virus(rAAV) has proven to be a promising gene delivery vector for human gene therapy. However, its application has been limited by difficulty in obtaining enough quantities of high-titer v...
关键词：RECOMBINANT ADENO-ASSOCIATED virus(rAAV)  RECOMBINANT HERPES simplex VIRUS type 1(rHSV-1)  vector  gene therapy  production system  green fluorescent protein(GFP). 
A novel method for purification of recombinant adeno-associated virus vectors on a large scale被引量：21
《Chinese Science Bulletin》2001年第6期485-489,共5页WU Xiaobing DONG Xiaoyan WU Zhijian CAO Hui NIU Dongbin QU Jianguo WANG Hong HOU Yunde 
the National High-technology Development Program (Grant No. Z20-05-02).
A novel method for recombinant adeno-associ-ated virus (rAAV) purification on large scale is described. The method involves three steps, including chloroform treatment, PEG/NaCl precipitation and chloroform extraction...
关键词：RECOMBINANT ADENO-ASSOCIATED virus CHLOROFORM purifi- cation. 
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