Results 1 to 10 of about 12,279,662 (388)

CRISPR Gene Therapy: Applications, Limitations, and Implications for the Future

Frontiers in Oncology, 2020
A series of recent discoveries harnessing the adaptive immune system of prokaryotes to perform targeted genome editing is having a transformative influence across the biological sciences. The discovery of Clustered Regularly Interspaced Short Palindromic
F. Uddin, C. Rudin, T. Sen
semanticscholar   +1 more source

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
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AAV-mediated pancreatic overexpression of Igf1 counteracts progression to autoimmune diabetes in mice

Molecular Metabolism, 2017
Objective: Type 1 diabetes is characterized by autoimmune destruction of β-cells leading to severe insulin deficiency. Although many improvements have been made in recent years, exogenous insulin therapy is still imperfect; new therapeutic approaches ...
Cristina Mallol, Estefania Casana, Veronica Jimenez, Alba Casellas, Virginia Haurigot, Claudia Jambrina, Victor Sacristan, Meritxell Morró, Judith Agudo, Laia Vilà, Fatima Bosch   +10 more
doaj   +1 more source

A comprehensive atlas of Aggrecan, Versican, Neurocan and Phosphacan expression across time in wildtype retina and in retinal degeneration

Scientific Reports, 2022
As photoreceptor cells die during retinal degeneration, the surrounding microenvironment undergoes significant changes that are increasingly recognized to play a prominent role in determining the efficacy of therapeutic interventions.
A. Matsuyama, A. A. Kalargyrou, A. J. Smith, R. R. Ali, R. A. Pearson   +4 more
doaj   +1 more source

Expression of Green Fluorescence Protein (GFP) in Zebrafish Muscle through Injection: A Gene Therapy Model [PDF]

, 2008
Expression of the target gene is important for gene therapy. Presently, localized transgenesis is used for gene therapy which can be achieved by a target gene expression. Here, we have reported the plasmid mediated gene therapy to zebrafish model.
C. H. Hsu, C. S. Lin, Chiranjib Chakraborty, Jhen Ru Shih, Z.H. Wen   +4 more
core   +2 more sources

Antibody-based CCR5 blockade protects Macaques from mucosal SHIV transmission

Nature Communications, 2021
CCR5 is a co-receptor for many transmitted HIV strains. Here, the authors show that biweekly injection of the CCR5-specific antibody Leronlimab protects rhesus macaques against infection following repeated intrarectal challenges of a CCR5-tropic SHIV.
Xiao L. Chang, Gabriela M. Webb, Helen L. Wu, Justin M. Greene, Shaheed Abdulhaqq, Katherine B. Bateman, Jason S. Reed, Cleiton Pessoa, Whitney C. Weber, Nicholas Maier, Glen M. Chew, Roxanne M. Gilbride, Lina Gao, Rebecca Agnor, Travis Giobbi, Jeffrey Torgerson, Don Siess, Nicole Burnett, Miranda Fischer, Oriene Shiel, Cassandra Moats, Bruce Patterson, Kush Dhody, Scott Kelly, Nader Pourhassan, Diogo M. Magnani, Jeremy Smedley, Benjamin N. Bimber, Nancy L. Haigwood, Scott G. Hansen, Timothy R. Brown, Lishomwa C. Ndhlovu, Jonah B. Sacha   +32 more
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Structural characterization of a novel human adeno-associated virus capsid with neurotropic properties

Nature Communications, 2020
Adeno-associated viruses (AAVs) are vehicles for gene therapy in humans, but currently only a limited amount of AAV serotypes is available. Here, the authors identify a novel AAV, AAVv66, and demonstrate enhanced production yields, virion stability, and ...
Hung-Lun Hsu, Alexander Brown, Anna B. Loveland, Anoushka Lotun, Meiyu Xu, Li Luo, Guangchao Xu, Jia Li, Lingzhi Ren, Qin Su, Dominic J. Gessler, Yuquan Wei, Phillip W. L. Tai, Andrei A. Korostelev, Guangping Gao   +14 more
doaj   +1 more source

The use of genes for performance enhancement: doping or therapy? [PDF]

, 2011
Recent biotechnological advances have permitted the manipulation of genetic sequences to treat several diseases in a process called gene therapy. However, the advance of gene therapy has opened the door to the possibility of using genetic manipulation ...
Collares, T. F., Collares, T. V., Oliveira, R. S., Seixas, F. K., Smith, Kevin R.   +4 more
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Modeling Anti-HIV-1 HSPC-Based Gene Therapy in Humanized Mice Previously Infected with HIV-1. [PDF]

, 2018
Investigations of anti-HIV-1 human hematopoietic stem/progenitor cell (HSPC)-based gene therapy have been performed by HIV-1 challenge after the engraftment of gene-modified HSPCs in humanized mouse models.
An, Dong Sung, Arokium, Hubert, Chen, Irvin SY, Cortado, Ruth, Jung, Yujin, Kamata, Masakazu, Khamaikawin, Wannisa, Kim, Patrick, Kim, Sanggu, Lam, Jennifer, Presson, Angela P, Shimizu, Saki, Wen, Jing, Xie, Yiming   +13 more
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The once and future gene therapy

Nature Communications, 2020
Gene therapy is at an inflection point. Recent successes in genetic medicine have paved the path for a broader second wave of therapies and laid the foundation for next-generation technologies. This comment summarizes recent advances and expectations for
Karen Bulaklak, C. Gersbach
semanticscholar   +1 more source