Results 41 to 50 of about 79,958 (292)

Immunity and AAV-mediated gene therapy for muscular dystrophies in large animal models and human trials

Frontiers in Microbiology, 2011
Adeno-associated viral (AAV) vector mediated gene replacement for the treatment of muscular dystrophy represents a promising therapeutic strategy in modern medicine.
Zejing eWang, Stephen J Tapscott, Stephen J Tapscott, Jeffrey S Chamberlain, Jeffrey S Chamberlain, Rainer eStorb, Rainer eStorb   +6 more
doaj   +1 more source

AAV-Tis21 infects DAOY and D283 MB cell lines and decreases Cyclin D1 mRNA expression levels in Tis21 dose-dependent manner.

, 2018
Real-time PCR analysis of mRNA obtained from DAOY (A and B) and D283 (C and D) cells infected with AAV overexpressing Tis21 (AAV-Tis21) or AAV-CBA (control). Increasing MOI employed for infection is indicated in graphs.
Manuela Ceccarelli (4398151), Giuliana Papoff (688457), Simone Samperna (828290), Felice Tirone (256018), Simonetta Santini (828289), Laura Micheli (256012), Cristiana Lalli (687846), Lorena Zentilin (1933051), Dario Presutti (828288), Giovina Ruberti (687850)   +9 more
core   +1 more source

GATA4‐Driven Transcription of HtrA1 Promotes Cellular Senescence in Ménière's Disease and Age‐Related Audio‐Vestibular Dysfunction

Advanced Science, EarlyView.
This study identifies the HDAC6/GATA4/HtrA1 axis as a critical driver of cellular senescence in the inner ear. GATA4 nuclear translocation, facilitated by HDAC6 downregulation, transcriptionally activates HtrA1, promoting hair cell senescence, SASP, and audio‐vestibular dysfunction in models of Ménière's disease and age‐related audio‐vestibular ...
Na Zhang, Na Li, Yan Wang, Jing Zhang, Jiahui Liu, Lei Chen, Yongdong Song, Yurong Mu, Yuechen Han, Yafeng Lyu, Xiaofei Li, Hanyue Wang, Jing Wang, Yao Lu, Zhaomin Fan, Daogong Zhang, Haibo Wang   +16 more
wiley   +1 more source

Distribution of nanoparticles throughout the cerebral cortex of rodents and non-human primates: Implications for gene and drug therapy

Frontiers in Neuroanatomy, 2014
When nanoparticles/proteins are infused into the brain, they are often transported to distal sites in a manner that is dependent both on the characteristics of the infusate and the region targeted. We have previously shown that AAV is disseminated within
Ernesto A Salegio, Ernesto A Salegio, Hillary eStreeter, Nikhil eDube, Piotr eHadaczek, Lluis eSamaranch, Adrian eKells, Waldy eSan Sebastián, Yuying eZhai, John eBringas, Ting eXu, John eForsayeth, Krystof S Bankiewicz   +12 more
doaj   +1 more source

AAV-ChR2 delivery to PVN neurons and optogenetic responses in the ARC.

, 2019
AAV-ChR2 was delivered by stereotaxic injection into the PVN and 8 weeks later, brain slices were stimulated by blue light to evaluate direct synaptic connectivity between PVN neurons and Kiss1 neurons in the arcuate. A. Expression of AAV-ChR2-GFP in PVN
Clemence Blouet (6519428), William Henry Colledge (476143), Victoria Kyle (6519425), Susan Jones (4079), Shel-Hwa Yeo (6519422)   +4 more
core   +1 more source

Development of a Rapid Adeno-Associated Virus (AAV) Identity Testing Platform through Comprehensive Mass Analysis of Full-length AAV Capsid Proteins

, 2023
Adeno-associated viruses (AAVs) are commonly used as vectors for the delivery of gene therapy targets. Characterization of AAV capsid proteins (VPs) and their post-translational modifications (PTMs) has become a critical attribute monitored to evaluate ...
Felipe, Guapo, Steven, Milian, Josh, Smith, Lisa, Strasser, Silvia, Millan-Martin, Richard, Snyder, Jonathan, Bones   +6 more
core   +1 more source

LC3B Mediated SETDB1‐Accounted Alcoholic Steatohepatitis via Lipidation‐Dependent LAP and Lipidation‐Independent Nuclear Stabilization

Advanced Science, EarlyView.
SETDB1 is progressively downregulated in ALD, correlating with disease severity. SETDB1 deficiency impairs LAP by disrupting Rubicon membrane localization, leading to defective lipid droplet clearance. Concurrently, loss of SETDB1 reduces nuclear LC3B, causing R‐loop accumulation and cGAS‐STING‐driven inflammation. Lipidated LC3B mediates LAP‐dependent
Yi Zhang, Tan Wei, Jiahang Wu, Chuixu Lin, Dongbo Zhu, Yanhui Li, Shuting Shi, Shishun Huang, Leiming Jiang, Hongzhi Wang, Meiqi Song, Pengfei Gao, Xu Wu, Mingjian Fan, Chaofeng Wei, Qian Wang, Lihui Qu, Zhigang Wang   +17 more
wiley   +1 more source

AAV vectors for FRET-based analysis of protein-protein interactions in photoreceptor outer segments

Frontiers in Neuroscience, 2016
Fluorescence resonance energy transfer (FRET) is a powerful method for the detection and quantification of stationary and dynamic protein-protein interactions.
Elvir Becirovic, Elvir Becirovic, Sybille Boehm, Sybille Boehm, Ong Nam Phuong Nguyen, Ong Nam Phuong Nguyen, Lisa Maria Riedmayr, Lisa Maria Riedmayr, Verena Hammelmann, Verena Hammelmann, Christian Schön, Christian Schön, Elisabeth Susanne Butz, Elisabeth Susanne Butz, Christian Wahl-Schott, Christian Wahl-Schott, Martin Biel, Martin Biel, Stylianos Michalakis, Stylianos Michalakis   +19 more
doaj   +1 more source

TRMT6‐Mediated m1A Modification of CDK9 mRNA Is a Dual‐Pronged Pathogenic Driver for HBV‐Related Hepatocellular Carcinoma

Advanced Science, EarlyView.
TRMT6‐mediated m1A modification in CDK9 mRNA enhances its mRNA stability and translation efficiency, thereby increasing the protein levels of CDK9. Upregulated CDK9 promotes the progression of HCC by elevating the levels of oncogenic factors including p‐STAT3, MCL1, and BCL‐2. On the other hand, CDK9 phosphorylates TARDBP at Ser254 to activate HBV core
Rui Zhang, Dandan Zong, Rui Liu, Yubo Wang, Qingqing Gu, Yao Yao, Wenfang Zheng, Mengmeng Yuan, Simeng Wang, Rongrong Cui, Daxu Li, Siwen Dang, Peng Hou   +12 more
wiley   +1 more source

Adeno-associated virus vector-based gene therapy for hereditary diseases: current problems of application and approaches to solve them

Биопрепараты: Профилактика, диагностика, лечение
INTRODICTION. Currently, gene therapy based on adeno-associated virus (AAV) vectors faces a  number of barriers, both biomedical and technological, which require studying and overcoming for further development of this gene therapy technology.AIM.
T. V. Egorova, A. A. Piskunov, D. A. Poteryaev   +2 more
doaj   +1 more source