Results 51 to 60 of about 74,668 (263)

Production of recombinant adeno-associated virus

, 2000
Currently, rAAV appears to be one of the most promising vectors for gene therapy applications. Attractive features of the vector include nonpathogenicity, the ability to infect nondividing cells, escape from host immune responses, and integration into the host genome.
G P, Gao, J M, Wilson, N A, Wivel
openaire   +2 more sources

Microglial Deubiquitinase OTUD7B Stabilizes STAT3 to Promote Neuroinflammation and Cognitive Decline in Alzheimer's Disease

Advanced Science, EarlyView.
. ABSTRACT Neuroinflammation driven by microglial activation is a defining feature of Alzheimer's disease (AD), yet the molecular mechanisms sustaining this proinflammatory state remain unclear. Here, we identify the deubiquitinase OTUD7B as a critical regulator of microglial activation and AD pathology.
Luyao Li, Hao Tang, Qin Yu, Yuyang Zhang, Binglu Shi, Ying Kong, Leiyu Xu, Jingjing Shao, Chenghong Hu, Lingyu She, Zhe Wang, Haiyi Chen, Aleksandr V. Samorodov, Gang Cao, Xia Zhao, Yi Wang   +15 more
wiley   +1 more source

Natural Adeno-Associated Virus Serotypes and Engineered Adeno-Associated Virus Capsid Variants: Tropism Differences and Mechanistic Insights

Viruses
Today, adeno-associated virus (AAV)-based vectors are arguably the most promising in vivo gene delivery vehicles for durable therapeutic gene expression. Advances in molecular engineering, high-throughput screening platforms, and computational techniques
Estrella Lopez-Gordo, Kyle Chamberlain, Jalish Mahmud Riyad, Erik Kohlbrenner, Thomas Weber   +4 more
doaj   +1 more source

Gm26550 Modulates Learning and Memory by Increasing IGF1 mRNA Expression and Stability in Nrf2−/− Mice

Advanced Science, EarlyView.
The present study shows that Nrf2 directly binds to the Gm26550 promoter, thereby activating Gm26550 transcription and increasing its expression. Mechanistically, Gm26550 promotes IGF1 expression by functionally antagonizing miR‐26a‐5p‐mediated repression and sequestering the RBP KHSRP, thereby enhancing hippocampal neuronal synaptic plasticity and ...
Hongfang Wang, Ziyao Wang, Dongyue Zuo, Zhaowen Su, Bowen Song, Jiamin Gao, Yizhou Zhang, Ruiting Zhao, Linjie Sun, Mengdi Li, Yirui Fan, Dandan Geng, Lei Wang   +12 more
wiley   +1 more source

AAV-based vectors for human diseases modeling in laboratory animals

Frontiers in Medicine
The development of therapeutic drugs and vaccines requires the availability of appropriate model animals that replicate the pathogenesis of human diseases. Both native and transgenic animals can be utilized as models.
Timur I. Aliev, Dmitry V. Yudkin
doaj   +1 more source

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

Potential for cellular stress response to hepatic factor VIII expression from AAV vector

Molecular Therapy: Methods & Clinical Development, 2016
Hemophilia A and B are coagulation disorders resulting from the loss of functional coagulation factor VIII (FVIII) or factor IX proteins, respectively.
Irene Zolotukhin, David M Markusic, Brett Palaschak, Brad E Hoffman, Meera A Srikanthan, Roland W Herzog   +5 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

Adeno-associated virus for cystic fibrosis gene therapy

Brazilian Journal of Medical and Biological Research, 2011
Gene therapy is an alternative treatment for genetic lung disease, especially monogenic disorders such as cystic fibrosis. Cystic fibrosis is a severe autosomal recessive disease affecting one in 2500 live births in the white population, caused by ...
S.V. Martini, P.R.M. Rocco, M.M. Morales
doaj   +3 more sources

Mucopolysaccharidosis type IIIB: a current review and exploration of the AAV therapy landscape

Neural Regeneration Research
Mucopolysaccharidoses type IIIB is a rare genetic disorder caused by mutations in the gene that encodes for N-acetyl-alpha-glucosaminidase. This results in the aggregation of heparan sulfate polysaccharides within cell lysosomes that leads to progressive
Courtney J Rouse, Victoria N Jensen, Coy D Heldermon   +2 more
doaj   +1 more source