GENE_THERAPY

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Innovative gene delivery systems for retinal disease therapy
《Neural Regeneration Research》2026年第2期542-552,共11页Hongguang Wu Ling Dong Shibo Jin Yongwang Zhao Lili Zhu 
Hongguang Wu,Both authors contributed equally to this work and share first authorship;Ling Dong,Both authors contributed equally to this work and share first authorship;。
The human retina,a complex and highly specialized structure,includes multiple cell types that work synergistically to generate and transmit visual signals.However,genetic predisposition or age-related degeneration can...
关键词：adeno-associated viruses delivery systems gene delivery gene therapy LENTIVIRUS nanoparticle delivery non-viral delivery retinal disease RETINA small molecular delivery 
Tropism-shifted AAV-PHP.eB-mediated bFGF gene therapy promotes varied neurorestoration after ischemic stroke in mice
《Neural Regeneration Research》2026年第2期704-714,共11页Rubing Shi Jing Ye Ze Liu Cheng Wang Shengju Wu Hui Shen Qian Suo Wanlu Li Xiaosong He Zhijun Zhang Yaohui Tang Guo-Yuan Yang Yongting Wang 
supported by the National Natural Science Foundation of China,Nos.81870921(to YW),81974179(to ZZ),82271320(to ZZ),82071284(to YT);National Key R&D Program of China,No.2022YFA1603600(to ZZ),2019YFA0112000(to YT);Scientific Research and Innovation Program of Shanghai Education Commission,No.2019-01-07-00-02-E00064(to GYY);Scientific and Technological Innovation Act Program of Shanghai Science and Technology Commission,No.20JC1411900(to GYY).
AAV-PHP.eB is an artificial adeno-associated virus(AAV)that crosses the blood-brain barrier and targets neurons more efficiently than other AAVs when administered systematically.While AAV-PHP.eB has been used in vario...
关键词：AAV-PHP.eB angiogenesis basic fibroblast growth factor gene therapy ischemic stroke Ly6A neurogenesis neurological function transient middle cerebral artery occlusion TROPISM 
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. 
Preclinical ex vivo IL2RG gene therapy using autologous hematopoietic stem cells as an effective and safe treatment for X-linked severe combined immunodeficiency disease
《Genes & Diseases》2025年第3期397-410,共14页Mingfeng Hu Qiling Xu Fang Zhang Karen F.Buckland Yelei Gao Weixia Du Yuan Ding Lina Zhou Xiulian Sun Lijia Ma Zhiyong Zhang Xuemei Tang Xiaodong Zhao Adrian J.Thrasher Yunfei An 
supported by the National Natural Science Foundation of China(No.82070135);the National Key R&D Program of China(No.2021YFC2700804);the CQMU Program for Youth Innovation in Future Medicine(China)(No.W0100).
X-linked severe combined immunodeficiency disease(X-SCID)is a rare inheriteddisease caused by mutations in the interleukin 2 receptor subunit gamma gene(IL2RG),whichencodes the common g chain protein,a subunit of the ...
关键词：Atypicaldiverse phenotype Gene therapy IL2RG Self-inactivating lentiviral vector X-linked severe combined immunodeficiency disease 
Repurposing Ancient Viral Tools for Precision Gene Therapy
《Bulletin of the Chinese Academy of Sciences》2025年第1期36-39,共4页YAN Fusheng 
The 2024 development of a precision-engineered retrotransposon system marked a significant milestone in mammalian genome-editing research.As appeared in the July 8 issue of Cell,this methodological breakthrough establ...
关键词：repurposing ancient viral tools site specific gene delivery ancient viral tools precision gene therapy retrotransposon system 
SP7 transcription factor ameliorates bone defect healing in low-density lipoprotein receptor-related protein 5(LRP5)-dependent osteoporosis mice
《Journal of Zhejiang University-Science B(Biomedicine & Biotechnology)》2025年第3期254-268,共15页Yue XI Qifeng JIANG Wei DAI Chaozhen CHEN Yang WANG Xiaoyan MIAO Kaichen LAI Zhiwei JIANG Guoli YANG Ying WANG 
Loss-of-function variants of low-density lipoprotein receptor-related protein 5(LRP5)can lead to reduced bone formation,culminating in diminished bone mass.Our previous study reported transcription factor osterix(SP7)...
关键词：Gene therapy Low-density lipoprotein receptor-related protein 5(LRP5) Transcription factor osterix(SP7) OSTEOPOROSIS Defect healing 
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