Results 71 to 80 of about 326,770 (247)

In Situ Genetic Engineering Facilitates MMP‐9‐Responsive TIMP1 Release Through Proteolytic Cleavage to Boost Diabetic Wound Healing

Advanced Science, EarlyView.
This study develops an MMP‐9‐responsive protein release system that transforms resident cells into drug depots through in situ genetic engineering. Anchored to the membrane, the therapeutic protein TIMP1 is selectively released upon MMP‐9 cleavage.
Yuan Zhang, Jiani Deng, Man Yee Cheung, Tingting Fu, Xufeng Qi, Dongqing Cai, Jianmin Sun, Gang Lu, Peng Shi, Wai‐Yee Chan, Xu Li, Hui Zhao   +11 more
wiley   +1 more source

Single‐Cell Dissection of Microglial Dysfunction in Diabetes‐Associated Neurodegenerative Vulnerability

Advanced Science, EarlyView.
Using single‐cell transcriptomic and epigenomic profiling, researchers mapped microglial dysfunction across the lifespan of db/db mice. The authors uncovered a nonlinear microglial remodeling featuring early inflammatory priming, a mid‐stage transcriptional inflection, and late dysfunction.
Jizhou Zhang, Tsz Kwan Yau, Joyce Tse, Tsz Ho Li, Dongjie Hu, Yong Juan Zhao, Ting‐Fung Chan   +6 more
wiley   +1 more source

Creation of a cardiotropic adeno-associated virus: the story of viral directed evolution

Virology Journal, 2013
Adeno-associated virus (AAV) is an important vector system for human gene therapy. Although use of AAV serotypes can result in efficient myocardial gene transfer, improvements in the transduction efficiency and specificity are still required. As a method
Yang Lin, Xiao Xiao
doaj   +1 more source

Synthetic Biology: Emerging Concepts to Design and Advance Adeno‐Associated Viral Vectors for Gene Therapy

Advanced Science, 2021
Three recent approvals and over 100 ongoing clinical trials make adeno‐associated virus (AAV)‐based vectors the leading gene delivery vehicles in gene therapy.
Hanna J. Wagner, Wilfried Weber, Martin Fussenegger   +2 more
doaj   +1 more source

Gestational Hypoxia Disrupts Medial Ganglionic Eminence Progenitor Dynamics and Interneuron Development in Schizophrenia

Advanced Science, EarlyView.
FES‐derived MGE spheroids exhibit progenitor‐stage alterations in developmental trajectory and hypoxia‐responsive transcriptional programs, followed by functional disruption. Gestational hypoxia recapitulates impaired progenitor proliferation, shortened cell‐cycle progression, interneuron developmental abnormalities, and schizophrenia‐like behaviors in
Peiyan Ni, Qiancheng Zhang, Youhui Jiang, Huiying Yao, Chuqing Zhou, Xiaoyi Tian, Shiyingnan Gao, Jun Zhao, Xinyi Ren, Naxin Xu, Mengshan Fu, Liansheng Zhao, Xueyu Qi, Xueli Yu, Wanjun Guo, Sangmi Chung, Fan Guo, Tao Li   +17 more
wiley   +1 more source

Directed evolution of novel AAV vectors for clinical gene therapy [PDF]

, 2022
Gene therapy – the delivery of genetic material to the cells of a patient for therapeutic benefit – has been increasingly successful in human clinical trials over the past decade, and there are numerous FDA-approved gene therapies.
Schaffer, David V
core   +1 more source

Development of a gene-activated matrix for enhanced AAV gene delivery in vitro

Frontiers in Bioengineering and Biotechnology
Recombinant AAV vectors are among the most extensively studied vectors for viral gene delivery due to their unique safety profile and their ability to mediate efficient, long-term transgene expression by persisting episomally in the nucleus.
Ahmed Musoski, Florent Poulhès, Cedric Sapet, Neelam Iqbal, Payal Ganguly, Payal Ganguly, Maximilian Kampick, Bastian Hoechst, Mario Marotta, Katja Dumler, Animesh Jha, Elena A. Jones, Peter V. Giannoudis, Peter V. Giannoudis, Marilys Blanchy, Percy Knolle, Olivier Zelphati, Martina Anton   +17 more
doaj   +1 more source

A Rare De Novo Missense Mutation in IFT122 Confers a Genetic Susceptibility Factor of Idiopathic Pediatric Uveitis Via Trio‐based Whole‐Exome Sequencing

Advanced Science, EarlyView.
A rare de novo IFT122‐A773E variant is identified in idiopathic pediatric uveitis and shown to exacerbate retinal inflammation and barrier dysfunction. Mechanistically, the variant enhances IFT43 interaction, elevates calcium signaling, and activates the MEK/ERK/FRA1 axis, revealing a previously unrecognized cilia‐associated pathway that may increase ...
Qian Zhou, Jiaxing Huang, Yilin Wang, Xingran Li, Xianyang Liu, Lingyu Dai, Hongshun Li, Qingfeng Wang, Jiangyi Liu, Guannan Su, Wanyun Zhang, Yang Deng, Qingfeng Cao, Yujie Lai, Xiang Luo, Changwei Huang, Ling Chen, Shengping Hou, Peizeng Yang   +18 more
wiley   +1 more source

Entorhinal Astrocyte Transplants Restore Spatial Exploration and Alleviate Amyloid‐Beta Pathology in Alzheimer's Mice

Advanced Science, EarlyView.
Impaired MEC astrocytic Ca2+ signaling is associated with fragmented spatial exploration in AD mice. Region‐specific glial progenitor transplantation generates engrafted astrocytes that are accompanied by improved AQP4 polarization, reduced amyloid‐β‐associated pathology, attenuated neuroinflammation, preserved synaptic integrity, and ameliorated ...
Fengjuan Wu, Jihua Fan, Xiang Zhou, Zhiqi Yang, Mingyue Gong, Mingzhu Huang, Zhenlu Cai, Shaofan Yang, Teng Teng, Chuanyan Yang, Jin Li, Haoyu Wang, Shuangshuang Dai, Chunhai Chen, Xiaowei Chen, Manxia Wang, Kuan Zhang   +16 more
wiley   +1 more source

Advances in Bioanalytical Techniques and Pharmacokinetics/Pharmacodynamics Research of Adeno-Associated Viral Vector Gene Therapy Drugs

罕见病研究
Recently, adeno-associated virus(AAV)vectors have been a hotspot in gene therapy and have shown great potential for rare diseases therapy, due to their low pathogenicity, mild immunogenicity and high tissue specificity.
REN Jianwei, ZHENG Xin, HAN Xiaohong
doaj   +1 more source