Results 111 to 120 of about 396,665 (375)

Anticancer Gene Transfer for Cancer Gene Therapy [PDF]

, 2014
Gene therapy vectors are among the treatments currently used to treat malignant tumors. Gene therapy vectors use a specific therapeutic transgene that causes death in cancer cells.
A Aiuti, A Aiuti, A Biffi, A Winkeler, AD Bucheit, AW Tong, B Everts, B Kaur, B Moss, B Vogelstein, BR Dix, C Alvarez-Breckenridge, C Heise, C Schliemann, C Springfeld, CC O’Shea, CE Gomez, CE Thomas, CJ Sherr, CM Lai, D Hanahan, D Meirow, D Moroziewicz, D Piwnica-Worms, DF Stojdl, DR Koehler, DR Koehler, E Kelly, EJ Boviatsis, F Simonelli, FJ Morling, GD Trobridge, HA Hoster, HD Klenk, HH Wong, I Friedrich, J Ansari, J Chou, J Cinatl Jr, J Clarke, J Foloppe, J Fueyo, J Hardcastle, J Jin, J Sinkovics, J Veerapong, JA Noser, JC Deutekom van, JM Bergelson, JM Markert, JN Harada, JR Bischoff, JW Bainbridge, K Boztug, K Hamada, K Iguchi, K Nakano, KD Smith, KW Peng, KW Peng, KW Peng, KW Reichard, KX Zhang, L Chen, L Yuan, M Ahonen, M Bergmann, M Chase, M Egeblad, M Kinali, M Sauane, MA Kay, MA Passini, MA Stoff-Khalili, ML Lamfers, MS Ferguson, N Cartier, N Cartier, N Natarajan, N Nayerossadat, NK Green, NL Fox, O Hallgren, O Meier, OG Donnelly, P Msaouel, P Msaouel, PA Lewitt, PG Spear, Q Wu, R Cattaneo, R Cattaneo, R Stripecke, R Waehler, RM Lorence, S Aggarwal, S Chalikonda, S Cirak, S Goverdhana, S Grimm, S Inoue, S Jones, S Kumar, S Nagano, S Suzuki, SA Vorburger, SK Pal, T Flotte, T Gianni, T Kuroda, T Mineta, T Muster, T Sasaki, T Todo, T Todo, T Todo, TC Doyle, TG Huang, WW Spurbeck, X Wang, Y Yi, YY Mahller, Z Peng, Z Zhang, ZS Guo, ZS Guo   +125 more
core   +1 more source

Adeno-associated virus serotype 9 antibodies in patients screened for treatment with onasemnogene abeparvovec

Molecular Therapy: Methods & Clinical Development, 2021
Spinal muscular atrophy is a progressive, recessively inherited monogenic neurologic disease, the genetic root cause of which is the absence of a functional survival motor neuron 1 gene. Onasemnogene abeparvovec (formerly AVXS-101) is an adeno-associated
John W. Day, Richard S. Finkel, Eugenio Mercuri, Kathryn J. Swoboda, Melissa Menier, Rudolf van Olden, Sitra Tauscher-Wisniewski, Jerry R. Mendell   +7 more
doaj  

Adeno-Associated Virus-Based Gene Therapy for CNS Diseases

Human Gene Therapy, 2016
Gene therapy is at the cusp of a revolution for treating a large spectrum of CNS disorders by providing a durable therapeutic protein via a single administration.
Michaël Hocquemiller, L. Giersch, M. Audrain, S. Parker, N. Cartier   +4 more
semanticscholar   +1 more source

Consequences and Mechanisms of Noise‐Induced Cochlear Synaptopathy and Hidden Hearing Loss, With Focuses on Signal Perception in Noise and Temporal Processing

Advanced Science, EarlyView.
Noise‐induced synaptopathy (NIS) is largely reversible due to self‐repair. NIS and noise‐induced hidden hearing loss are two concepts with similarities and differences. The major hearing deficits in NIHHL are temporal processing disorders. The translation of animal data in NIS studies to humans is hindered by many factors.
Hui Wang, Steven J Aiken, Jian Wang
wiley   +1 more source

Gene-edited pseudogene resurrection corrects p47phox-deficient chronic granulomatous disease

Blood Advances, 2017
: Pseudogenes are duplicated genes with mutations rendering them nonfunctional. For single-gene disorders with homologous pseudogenes, the pseudogene might be a target for genetic correction.
Randall K. Merling, Douglas B. Kuhns, Colin L. Sweeney, Xiaolin Wu, Sandra Burkett, Jessica Chu, Janet Lee, Sherry Koontz, Giovanni Di Pasquale, Sandra A. Afione, John A. Chiorini, Elizabeth M. Kang, Uimook Choi, Suk See De Ravin, Harry L. Malech   +14 more
doaj  

Dosage Thresholds and Influence of Transgene Cassette in Adeno-Associated Virus-Related Toxicity.

Human Gene Therapy, 2018
Today, there are >500 published studies and 40 clinical trials to treat retinal disorders using gene therapy. The great majority of them rely on the use of adeno-associated virus vectors (AAV) for therapeutic gene delivery.
Hanen Khabou, Chloé Cordeau, Laure Pacot, S. Fisson, D. Dalkara   +4 more
semanticscholar   +1 more source

Characterization of the Rep78/Adeno-Associated Virus Complex

Virology, 1997
Adeno-associated virus (AAV) replication proteins Rep78 and Rep68 play major roles in the life cycle of AAV. We have recently provided in vivo evidence for the existence of a covalent association between Rep78 and virion single-stranded (ss) AAV DNA (K. M. R. Prasad and J.P. Trempe(1995) Virology 214, 360-370).
Changhong Zhou, James P. Trempe, Konkal Matt Renuka Prasad   +2 more
openaire   +3 more sources

Long‐Lasting Auditory and Vestibular Recovery Following Gene Replacement Therapy in a Novel Usher Syndrome Type 1c Mouse Model

Advanced Science, EarlyView.
This study shows that gene replacement therapy using the AAV2/Anc80L65 virus can successfully restore hearing and balance in Ush1c knockout mice. The treatment leads to lasting improvements in both auditory and vestibular functions, highlighting its potential as a therapeutic approach for genetic hearing loss and vestibular disorders in humans ...
Weinan Du, Jun Huang, Aizhen Zhang, Fangfang Zhao, Tianwen Chen, Quinn M. McDermott, Tony Zheng, Haibo Wang, Rongli Zhang, Xiaolin Zhang, Jerome Allison, Hong Zhu, Wu Zhou, Qing Yin Zheng   +13 more
wiley   +1 more source

Adeno‐associated virus vectors for gene therapy—focusing on melanoma

Interdisciplinary Medicine
Adeno‐Associated Virus (AAV) vectors have been found to have great potential in the field of gene therapy due to their unique properties. These nonpathogenic vectors exhibit high tissue specificity, low immunogenicity, and sustained gene expression ...
Xingyue Wang, Miao Wei, Rui Miao, Xiujing Hao, Min Li, Wenxin Wang, Zhonglei He   +6 more
doaj   +1 more source

Muscle‐Derived Small Extracellular Vesicles Mediate Exercise‐Induced Cognitive Protection in Chronic Cerebral Hypoperfusion

Advanced Science, EarlyView.
sEVs have a critical role in orchestrating interorgan crosstalk and mediating exercise‐induced therapeutic effects. Lin et al. demonstrates that sEVs miR‐17/20a‐5p mediates the muscle‐brain crosstalk and emphasizes the central role of mTOR signaling in executing molecular programs that can protect brain health in response to exercise. Abstract Physical
Huawei Lin, Lianhua Yin, Weilin Liu, Rui Li, Tao Jiang, Minguang Yang, Yajun Cao, Sinuo Wang, Yan Yu, Cong Chen, Xiaoqin Guo, Wenju Wang, Huanhuan Liu, Yaling Dai, Jiamin Yan, Yanting Lin, Yanyi Ding, Chendong Ruan, Lei Yang, Tiecheng Wu, Jing Tao, Lidian Chen   +21 more
wiley   +1 more source