Results 141 to 150 of about 214,719 (303)

Adeno-associated virus mediated expression of human erythropoietin in vitro

, 2002
AIM: To investigate the expression of human erythropoietin (hEpo) in 293 cell line using adeno-associated virus (AAV) vector, and provide information for its potential application in human gene therapy.
Zhao, XC, Diao, Y, Wang, GJ, Xu, RA
core  

LDLR-Gene therapy for familial hypercholesterolaemia: Problems, progress, and perspectives

, 2010
Coronary artery diseases (CAD) inflict a heavy economical and social burden on most populations and contribute significantly to their morbidity and mortality rates.
Charles Coutelle, Harbottle, R, Anna L David, Themis, M, Al-Allaf, FA, Waddington, SN, David, A, Faisal A Al-Allaf, Michael Themis, Coutelle, C, Simon N Waddington, Richard Harbottle   +11 more
core   +1 more source

The Dynamics of Neurofilament Light Chain in Spinal Muscular Atrophy

Annals of Neurology, EarlyView.
Objective Newborn screening (NBS) for spinal muscular atrophy (SMA) facilitates early diagnosis and treatment for affected individuals. However, fluid biomarkers that provide early insights into disease activity and outcomes in a neonatal cohort and those unable to access (due to reimbursement criteria) or deferring immediate treatment are lacking ...
Arlene D'Silva, Karen Herbert, Lakshmi Balaji, Jia Mei He, Tejaswi Kandula, Hugo A. Sampaio, Hooi‐Ling Teoh, Esther Tantsis, Jihee Sohn, Nancy Briggs, Nickson Ning, Matthew C. Kiernan, Didu S. Kariyawasam, Michelle A. Farrar   +13 more
wiley   +1 more source

Nonviral Gold Nanoparticle‐Mediated Delivery of CRISPR‐Cas9 Ribonucleoprotein and Long DNA Transgenes Into Primary Blood Cells

Advanced NanoBiomed Research, EarlyView.
CRISPR/Cas9 has revolutionized the field of gene therapy, but delivery remains an outstanding issue. We propose a nonviral gold‐nanoparticle platform for co‐delivery of CRISPR/Cas9 ribonucleoprotein and long 2.1 kilobase dsDNA transgene constructs. This CRISPR‐AuNP is inexpensive to produce and mediate gene editing and DNA delivery in T cells and CD34+
Rachel A. Cunningham, Karthikeya S. V. Gottimukkala, Daniel D. Lane, Katrina Poljakov, Patricia Lipson, Mark R. Enstrom, Alessandro Rizzi, Aude G. Chapuis, Jennifer E. Adair   +8 more
wiley   +1 more source

Adeno-Associated Virus Engineering and Load Strategy for Tropism Modification, Immune Evasion and Enhanced Transgene Expression

Xun Zhou,1,2,* Jingzhou Liu,2,* Shuang Xiao,2,3,* Xiaoqing Liang,1,2 Yi Li,2 Fengzhen Mo,3 Xin Xin,2 Yang Yang,2 Chunsheng Gao1,2 1School of Pharmacy, Henan University, Kaifeng, People’s Republic of China; 2State Key Laboratory of Toxicology ...
Liu J, Li Y, Yang Y, Liang X, Mo F, Xin X, Zhou X, Xiao S, Gao C   +8 more
core  

Modified Adeno-associated virus (AAV) particles for gene therapy

, 2019
The present invention relates to improved adeno-associated virus (AAV) particles for gene delivery and gene therapy. Provided are adeno associated virus (AAV) particles that comprise a modified capsid. The present invention further relates to methods for
Heppenstall, P., Maffei, M., de Castro Reis, Fernanda, Pouw, K. M.   +3 more
core  

Novel assay format for totalanti-adeno-associated virus antibodydetection with low capsid consumption andbuilt-in specificity control: supplementary data

Aim: To develop an assay format for detection of total anti-adeno–associated virus 2 (AAV2) antibodieswith low capsid material consumption. Methods: An immune complex (IC) assay format was developed.The format is based on the formation of ICs in solution
Kay Stubenrauch (10832767), Uwe Wessels (18175951), Klaus Mayer (57567), Julia Fakhiri (18175952), Florian Neff (14944185), Ulrich Brinkmann (279764)   +5 more
core   +1 more source

Adeno-associated virus (AAV)-mediated suppression of Ca2+/calmodulin kinase IV activity in the nucleus accumbens modulates emotional behaviour in mice-2

, 2011
Copyright information:Taken from "Adeno-associated virus (AAV)-mediated suppression of Ca2+/calmodulin kinase IV activity in the nucleus accumbens modulates emotional behaviour in mice"http://www.biomedcentral.com/1471-2202/8/105BMC Neuroscience 2007;8():
Hilmar Bading (78535), Miriam Schneider (85342), Sheng-Jia Zhang (85344), Matthias Klugmann (85345), Rainer Spanagel (85343)   +4 more
core   +1 more source

Comparison of Receptor‐Mediated Endocytosis and Its Application to Enhance DNA Transfection by TFAMoplex

Advanced NanoBiomed Research, EarlyView.
We developed an assay to distinguish cellular binding from internalization. Compatible with microscopy and high‐throughput screening, the method identifies ligand‐mediated uptake. Applying top candidates to a protein‐based DNA carrier enhanced transfection efficiency, providing a rational strategy to improve non‐viral gene delivery systems.
David Scherer, Steffen Honrath, Jonas Kurmann, Alexander Klipp, Finn Brigger, Jean‐Christophe Leroux   +5 more
wiley   +1 more source

Improved Detection of Myositis‐Specific Autoantibodies Using Luciferase Immunoprecipitation Systems Assay: Comparison With Line Blot and Conventional Immunoprecipitation

Arthritis &Rheumatology, EarlyView.
Objective Myositis‐specific autoantibodies guide the diagnosis and classification of idiopathic inflammatory myopathies, but current immunoassays vary in accuracy, particularly for autoantibodies associated with interstitial lung disease. To compare the performance of the luciferase immunoprecipitation systems (LIPS) assay with line blot and ...
Peter D. Burbelo, Iago Pinal‐Fernandez, Maria Casal‐Dominguez, Xin Tian, Shijinqiu Gao, Neha Gupta, Eric W. Robbins, John A. Chiorini, Christopher A. Mecoli, Lisa Christopher‐Stine, Livia Casciola‐Rosen, Andrew L. Mammen, Sonye K. Danoff, Anthony F. Suffredini, Julio A. Huapaya   +14 more
wiley   +1 more source