Results 11 to 20 of about 1,824,389 (266)

Gene therapy [PDF]

Current Problems in Surgery, 1996
Many advances have been made in the study of genetics over the past decade. The ultimate goal of this research is to develop medical applications which would provide a cure for the many fatal genetically linked diseases to which conventional medicine has
Danaher, Kerry
core   +4 more sources

Is it ‘gene therapy’? [PDF]

Journal of Law and the Biosciences, 2018
What, exactly, is "gene therapy"? Crystallizing a modern definition of "gene therapy" has now become critically important on several fronts. Following the recent of passage the 21st Century Cures Act, FDA now relies on the term to give regulatory incentives to sponsors of certain ‘regenerative medicines’, including ‘gene therapies’.
Jacob S. Sherkow, Jacob S. Sherkow, Jacob S. Sherkow, Patricia J. Zettler, Henry T. Greely   +4 more
openaire   +3 more sources

Gene therapy [PDF]

Proceedings of the National Academy of Sciences, 1997
In recent years, there have been a number of technological breakthroughs that have allowed for clinical trials in gene therapy to be initiated. In combination with the genome initiative, the potential for new therapeutics is limitless. Although an enormous amount of information has been obtained in a relatively short period of time, gene therapy ...
Mark A. Kay, Peter M. Hoogerbrugge, Dexi Liu   +2 more
openaire   +3 more sources

Gene therapy on the move [PDF]

EMBO Molecular Medicine, 2013
The first gene therapy clinical trials were initiated more than two decades ago. In the early days, gene therapy shared the fate of many experimental medicine approaches and was impeded by the occurrence of severe side effects in a few treated patients.
Axel Schambach, Axel Schambach, Kerstin B. Kaufmann, Anne Galy, Hildegard Büning, Manuel Grez   +5 more
openaire   +7 more sources

Gene therapy. [PDF]

Gut, 1982
Gene therapy is not yet possible, but may become feasible soon, particularly for well understood gene defects. Although treatment of a patient raises no ethical problems once it can be done well, changing the genes of an early embryo is more difficult, controversial and unlikely to be required clinically.
openaire   +4 more sources

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

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

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
doaj   +1 more source

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