Results 1 to 10 of about 1,824,339 (223)
Stochastic models and numerical algorithms for a class of regulatory gene networks [PDF]
arXiv, 2008 Regulatory gene networks contain generic modules like those involving feedback loops, which are essential for the regulation of many biological functions. We consider a class of self-regulated genes which are the building blocks of many regulatory gene networks, and study the steady state distributions of the associated Gillespie algorithm by providing
Fournier, Thomas +5 more
arxiv +4 more sources
Nature Communications, 2021 Mucopolysaccharidosis type IVA (MPSIVA) is a lysosomal storage disorder causing severe skeletal and non-skeletal alterations in patients. Here, the authors generate a MPSIVA rat model that mimics the disabling human pathology and develop an AAV9-Galns ...
Joan Bertolin +18 more
doaj +1 more source
Optimized riboswitch-regulated AAV vector for VEGF-B gene therapy
Frontiers in Medicine, 2022 Gene therapy would greatly benefit from a method to regulate therapeutic gene expression temporally. Riboswitches are small RNA elements that have been studied for their potential use in turning transgene expression on or off by ligand binding.
Reetta A. E. Eriksson +16 more
doaj +1 more source
Cell & Bioscience, 2022 Background Bile acid (BA) homeostasis is mainly regulated by bile salt excretory pump (BSEP), a hepatocyte transporter that transfers BAs to the bile.
Javier Martínez-García +6 more
doaj +1 more source
Gene conversion following CRISPR/Cas9 DNA cleavage: an overlooked effect [PDF]
, 2021 In the last few years the rapid pace in the development of gene editing technology has sparked hopes for curing rare diseases, leading to a potentially transformative impact in the field of gene therapy.GB receives funding from the National collaborative
Burgio, Gaetan
core +1 more source
Gene Therapy for Cardiovascular Disease [PDF]
, 2003 The last decade has seen substantial advances in the development of gene therapy strategies and vector technology for the treatment of a diverse number of diseases, with a view to translating the successes observed in animal models into the clinic ...
Baker, Andrew H. +3 more
core +9 more sources
Scientific Reports, 2023 Obesity and type 2 diabetes are two closely related diseases representing a serious threat worldwide. An increase in metabolic rate through enhancement of non-shivering thermogenesis in adipose tissue may represent a potential therapeutic strategy ...
Jordi Rodó +9 more
doaj +1 more source
Exosome-mediated stable epigenetic repression of HIV-1
Nature Communications, 2021 A strategy to control HIV-1 infection is to stably repress HIV-1 and induce “deep latency”. Here the authors show that a recombinant anti-HIV-1-1 protein can be packaged as mRNA into exosomes and delivered systemically to repress HIV-1-1 within the ...
Surya Shrivastava +7 more
doaj +1 more source
Hepatocyte growth factor gene therapy reduces ventricular arrhythmia in animal models of myocardial ischemia. [PDF]
, 2005 It was recently reported that gene therapy using hepatocyte growth factor (HGF) has the potential to preserve cardiac function after myocardial ischemia. We speculated that this HGF gene therapy could also prevent ventricular arrhythmia.
Aoki, Motokuni +7 more
core +1 more source
Molecular Therapy: Methods & Clinical Development, 2016 Recombinant adeno-associated viral (rAAV) vectors containing oversized genomes provide transgene expression despite low efficiency packaging of complete genomes.
Sirkka Kyostio-Moore +9 more
doaj +1 more source