GENE_THERAPY

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The Citron homology domain of MAP4Ks improves outcomes of traumatic brain injury
《Neural Regeneration Research》2025年第11期3233-3244,共12页Xiaoling Zhong Wenjiao Tai Meng-Lu Liu Shuaipeng Ma Tianjin Shen Yuhua Zou Chun-Li Zhang 
supported by the TARCC,Welch Foundation Award(I-1724);the Decherd Foundation;the Pape Adams Foundation,NIH grants NS092616,NS127375,NS117065,NS111776。
The mitogen-activated protein kinase kinase kinase kinases(MAP4Ks)signaling pathway plays a pivotal role in axonal regrowth and neuronal degeneration following insults.Whether targeting this pathway is beneficial to b...
关键词：adeno-associated virus Citron homology Citron homology domain gene therapy mitogen-activated protein kinase kinase kinase kinases traumatic brain injury 
Recovery of the injured neural system through gene delivery to surviving neurons in Parkinson’s disease
《Neural Regeneration Research》2025年第10期2855-2861,共7页Chanchal Sharma Sehwan Kim Hyemi Eo Sang Ryong Kim 
supported by the National Research Foundation of Korea(RS-2023-00245298);the Korea Healthcare Technology R&D(HI21C1795)grants,funded by the Korean government(to SRK).
A critical unaddressed problem in Parkinson’s disease is the lack of therapy that slows or hampers neurodegeneration.While medications effectively manage symptoms,they offer no long-term benefit because they fail to ...
关键词：adeno-associated virus gene therapy neuroprotection neurorestoration neurotrophic factor nigrostriatal dopamine pathway pro-survival protein 
AAV2-PDE6B restores retinal structure and function in the retinal degeneration 10 mouse model of retinitis pigmentosa by promoting phototransduction and inhibiting apoptosis
《Neural Regeneration Research》2025年第8期2408-2419,共12页Ruiqi Qiu Mingzhu Yang Xiuxiu Jin Jingyang Liu Weiping Wang Xiaoli Zhang Jinfeng Han Bo Lei 
supported by the National Natural Science Foundation of China,Nos.82071008(to BL)and 82004001(to XJ);Medical Science and Technology Program of Health Commission of Henan Province,No.LHGJ20210072(to RQ);Science and Technology Department of Henan Province,No.212102310307(to XJ)。
Retinitis pigmentosa is a group of inherited diseases that lead to retinal degeneration and photoreceptor cell death.However,there is no effective treatment for retinitis pigmentosa caused by PDE6B mutation.Adeno-asso...
关键词：APOPTOSIS AAV2-PDE6B ERK1/2 gene therapy PHOTOTRANSDUCTION PROTEOMICS rd10 retinitis pigmentosa 
Gene therapy for spinal muscular atrophy:perspectives on the possibility of optimizing SMN1 delivery to correct all neurological and systemic perturbations
《Neural Regeneration Research》2025年第7期2011-2012,共2页Sharon J.Brown Rafael J.Yáñez-Muñoz Heidi R.Fuller 
supported by the Faculty Research Fund(Faculty of Medicine&Health Science,Keele University)Career Development Award–(April 2022)(to SJB)。
Spinal muscular atrophy(SMA)is a genetic condition that results in selective lower motor neuron loss with concomitant muscle weakness and atrophy.The genetic cause of SMA was understood in 1995 when loss or impairment...
关键词：ATROPHY traditionally absolute 
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. 
Monogenic gene therapy for glaucoma and optic nerve injury
《Neural Regeneration Research》2025年第3期815-816,共2页Chikako Harada Xiaoli Guo Takayuki Harada 
supported by Japan Society for the Promotion of Science (JSPS) KAKENHI Grants-in-Aid for Scientific Research (JP22K09804 to CH;JP21K09688 to XG;JP19KK0229, JP21H04786, JP21H02819 and JP21K18279 to TH);the Shiseido Female Researcher Science Grant (to XG);Mitsubishi Foundation;Takeda Science Foundation (to TH)。
The prevalence of glaucoma, the second leading cause of global blindness, is increasing due to aging populations. In glaucoma, degeneration of the optic nerve and retinal ganglion cells(RGCs) causes visual field defec...
关键词：VISUAL GLAUCOMA DEGENERATION 
The emerging role of nitric oxide in the synaptic dysfunction of vascular dementia
《Neural Regeneration Research》2025年第2期402-415,共14页Xiaorong Zhang Zhiying Chen Yinyi Xiong Qin Zhou Ling-Qiang Zhu Dan Liu 
supported by the National Key R&D Program of China,No.2019YFE0121200(to LQZ);the National Natural Science Foundation of China,Nos.82325017(to LQZ),82030032(to LQZ),82261138555(to DL);the Natural Science Foundation of Hubei Province,No.2022CFA004(to LQZ);the Natural Science Foundation of Jiangxi Province,No.20224BAB206040(to XZ);Research Project of Cognitive Science and Transdisciplinary Studies Center of Jiangxi Province,No.RZYB202201(to XZ).
With an increase in global aging,the number of people affected by cerebrovascular diseases is also increasing,and the incidence of vascular dementia-closely related to cerebrovascular risk-is increasing at an epidemic...
关键词：endoplasmic reticulum stress endothelial nitric oxide synthase gene therapy nitric oxide NO-sGC-cGMP pathway synaptic dysfunction vascular dementia 
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 
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 
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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