Results 41 to 50 of about 199,900 (336)

Toward Personalized Gene Therapy: Characterizing the Host Genetic Control of Lentiviral-Vector-Mediated Hepatic Gene Delivery

Molecular Therapy: Methods & Clinical Development, 2017
The success of lentiviral vectors in curing fatal genetic and acquired diseases has opened a new era in human gene therapy. However, variability in the efficacy and safety of this therapeutic approach has been reported in human patients.
Thipparat Suwanmanee, Martin T. Ferris, Peirong Hu, Tong Gui, Stephanie A. Montgomery, Fernando Pardo-Manuel de Villena, Tal Kafri   +6 more
doaj   +1 more source

Exploration of high-efficiency transfection methods for sheep fibroblasts OAR-L1

浙江大学学报. 农业与生命科学版, 2022
In order to achieve high-efficiency expression of exogenous protein in sheep lung fibroblasts OAR-L1, and to explore a suitable transfection method for the cell line, the transfection efficiencies of polyethyleneimine (PEI), LipofectamineTM 2000 ...
WU Fei, WU Jie, CHEN Xueqiu, ZHOU Jingru, ZHANG Hui, HUANG Yan, SHI Hengzhi, YANG Yi, MA Guangxu, DU Aifang   +9 more
doaj   +1 more source

Cardiac-derived CTRP9 protects against myocardial ischemia/reperfusion injury via calreticulin-dependent inhibition of apoptosis. [PDF]

, 2018
Cardiokines play an essential role in maintaining normal cardiac functions and responding to acute myocardial injury. Studies have demonstrated the heart itself is a significant source of C1q/TNF-related protein 9 (CTRP9). However, the biological role of
Feng, Pan, Gao, Erhe, Lau, Wayne Bond, Ma, Xin-Liang, Qin, Zhigang, Sun, Yang, Tan, Yanzhen, Xu, Xuezeng, Yang, Jian, Yi, Dinghua, Yi, Wei, Yu, Shiqiang, Zhang, Zhengbin, Zhao, Dajun   +13 more
core   +2 more sources

FIV: from lentivirus to lentivector [PDF]

The Journal of Gene Medicine, 2004
Molecular virological understanding of the feline immunodeficiency virus (FIV) life cycle is increasing, facilitating rational derivation of improved vectors from this non-primate lentivirus. The packaging signal has been mapped, a central DNA flap has been identified, and class I integrase mutants have been validated.
Dyana T, Saenz, Eric M, Poeschla
openaire   +2 more sources

Gene therapy for the neurological manifestations in lysosomal storage disorders

Journal of Lipid Research, 2014
Over the past several years, considerable progress has been made in the development of gene therapy as a therapeutic strategy for a variety of inherited metabolic diseases, including neuropathic lysosomal storage disorders (LSDs).
Seng H. Cheng
doaj   +1 more source

CRISPR-Cas9 screens in human cells and primary neurons identify modifiers of C9ORF72 dipeptide-repeat-protein toxicity. [PDF]

, 2018
Hexanucleotide-repeat expansions in the C9ORF72 gene are the most common cause of amyotrophic lateral sclerosis and frontotemporal dementia (c9ALS/FTD).
A Berson, A Jovičić, AA Dominguez, Aaron D. Gitler, AC Elden, AD Gitler, AD Gitler, AD Gitler, AE Renton, AM Jablonski, Amy Li, Ana Jovičić, AS Ng, BD Freibaum, C Sidrauski, C Sidrauski, C. Kimberly Tsui, D Ron, David W. Morgens, DW Morgens, DW Morgens, E Yeger-Lotem, EM Lynes, EY Son, FB Gao, Gregor Bieri, H Koike-Yusa, HJ Kim, HJ Kim, I Kwon, IR Mackenzie, IR Mackenzie, J Couthouis, J Joung, James Ousey, JC Christianson, JG Wideman, JP Taylor, Julien Couthouis, Justin K. Ichida, K Kanekura, K Mori, K Zhang, KH Lee, KM Green, KY Shi, L Ellgaard, L Huang, LA Becker, LA Gilbert, LA Gilbert, M Armakola, M DeJesus-Hernandez, M Neumann, M Prudencio, MA Horlbeck, Marc Tessier-Lavigne, MC Jonikas, MH Schludi, MI Love, Michael C. Bassik, Michael S. Haney, NF Liachko, Nicholas J. Kramer, Nicholas T. Hertz, O Shalem, OM Peters, PE Ash, Q Zhou, R Dafinca, R Lopez-Gonzalez, R Seijffers, RJ Platt, RM Deans, Rosanna Ma, S Boeynaems, S Boeynaems, S Ju, S Konermann, S Mizielinska, S Treusch, SC Ling, SJ Zhang, T Wang, T Westergard, T Zu, TF Gendron, W Cheng, X Wen, Y Lin, Y Zhou, Yingxiao Shi, YJ Zhang, YS Davidson, Z Shevtsova, Z Sun   +95 more
core   +1 more source

Are Lentivirus Vectors Safer?

Molecular Therapy, 2007
In 2003, the field of gene therapy faced serious adverse events, apparently arising in part from insertional activation of the LMO2 gene in a clinical gene therapy trial in Paris that sought to correct deficiency of the interleukin-2 common -chain receptor (IL2RG).
openaire   +2 more sources

Temporal proteomic analysis of HIV infection reveals remodelling of the host phosphoproteome by lentiviral Vif variants

eLife, 2016
Viruses manipulate host factors to enhance their replication and evade cellular restriction. We used multiplex tandem mass tag (TMT)-based whole cell proteomics to perform a comprehensive time course analysis of >6500 viral and cellular proteins ...
Edward JD Greenwood, Nicholas J Matheson, Kim Wals, Dick JH van den Boomen, Robin Antrobus, James C Williamson, Paul J Lehner   +6 more
doaj   +1 more source

Intramuscular Delivery of Gene Therapy for Targeting the Nervous System

Frontiers in Molecular Neuroscience, 2020
Virus-mediated gene therapy has the potential to deliver exogenous genetic material into specific cell types to promote survival and counteract disease.
Andrew P. Tosolini, James N. Sleigh, James N. Sleigh   +2 more
doaj   +1 more source

Highly Efficient Gene Expression in B Lymphocytes Mediating by Lentivirus Vector [PDF]

, 2012
Gene transduction and expression efficiencies among several type cell lines were compared by using vesicular stomatitis virus-glycoprotein (VSV-G) pseudotyped human immunodeficiency virus type-1 (HIV-1) based lentivirus vector. Large discrepancies of the
Akira Tempaku
core   +1 more source