Results 41 to 50 of about 89,643 (298)

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

Human iPSC‐Derived Tissue‐Engineered Nigrostriatal Pathway for Chronic Restoration of Dopaminergic Axon Tracts in a Rat Parkinson's Model

Advanced Healthcare Materials, EarlyView.
Inspired by conventional cell therapy and emerging bioengineering approaches, the tissue‐engineered nigrostriatal pathway (TE‐NSP) has an anatomically‐inspired design comprising a population of human dopaminergic neurons with unidirectional, long‐projecting axon tracts.
Wisberty J. Gordián‐Vélez, Dimple Chouhan, Kevin D. Browne, James Lim, Saarang P. Karandikar, Rodrigo A. España, John E. Duda, H. Isaac Chen, D. Kacy Cullen   +8 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

Syngeneic AAV Pseudo-particles Potentiate Gene Transduction of AAV Vectors [PDF]

, 2017
Adeno-associated virus (AAV) vectors have emerged as a safe and efficient gene therapy platform. One complication is that a significant amount of empty particles have always been generated as impurities during AAV vector production.
Ruian Xu, Biao Dong, Firrman|, Qizhao Wang, Liu, LinShu, Diao, Yong, Xiao|, LinShu Liu, Pokiniewski, Katie A., Wu, Zhongren, Mario P.S. Chin, Xiongwen Chen, Dong, Biao, Yong Diao, Chen, Xiongwen, Katie A. Pokiniewski, Zhongren Wu, Chen|, Weidong Xiao, Xu, Ruian, Jenni Firrman, Wang, Qizhao, Firrman, Jenni, Chin, Mario P. S., XIAO, WEIDONG   +24 more
core   +1 more source

Rab1A Promotes Hepatic Steatosis by Suppressing Mitophagy via the Raf‐1/ERK1/2/PINK1 Signaling Axis

Advanced Science, EarlyView.
Why does fat accumulate in the liver? Our study reveals Rab1A as the molecular switch that silences the cell's mitochondrial cleanup crew. Turning Rab1A off reactivates mitophagy, clears excess fat, and reverses fatty liver disease in mice—offering a promising new therapeutic target for MASLD, a growing global health challenge.
Li Zhang, Jianhua Li, Huilu Zhang, Qi Qin, Yichen Huang, Xiaofan Tian, Chao Sun, Binbin Li, Zhengxin Wang, Xin Zhang   +9 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

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

Gonadotropin‐Inhibitory Hormone (GnIH) and Its Receptor GPR147 Mediates Acute Stress‐Induced Activation of the Locus Coeruleus‐SAM Axis

Advanced Science, EarlyView.
ABSTRACT Gonadotropin‐inhibitory hormone (GnIH, also known as RFamide‐related peptide‐3, RFRP‐3) is a hypothalamic neuropeptide that regulates vertebrate reproduction via its receptor GPR147. Although GnIH is known to mediate stress‐induced reproductive suppression, its role in broader stress responses remains unclear.
Jiani Zhang, Hongyu Zhu, Wenqi Wang, Bingqian Shen, Yuanyuan Xin, Xingxing Song, Xuanyi Lu, Lixuan Li, Siting Chen, Xiaochen Zhang, Jialing Zeng, Haodong Zheng, Jianlong Lao, Ke Peng, Yixian Wei, Jiaming Zheng, Chuanhuo Hu, Xiaoye Wang, Xun Li   +18 more
wiley   +1 more source

Chem-AAV: Chemically Modified AAV for Gene Therapy

, 2017
Événement(s) lié(s) : - (ASGCT); Washington, DC (USA) - (2017-05-10 - 2017-05-13)Chem-AAV: Chemically Modified AAV for Gene Therapy.
Toublanc, Estelle, Gouin, Sebastien, Bouzelha, Mohammed, Ayuso, Eduard, Mevel, Mathieu, Blouin, Veronique, Deniaud, David, Dubreil, Laurence, Moullier, Philippe   +8 more
core   +3 more sources

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