Magnetic Field-Assisted Gene Delivery: Achievements and Therapeutic Potential [PDF]
arXiv, 2011 The discovery in the early 2000's that magnetic nanoparticles (MNPs) complexed to nonviral or viral vectors can, in the presence of an external magnetic field, greatly enhance gene transfer into cells has raised much interest. This technique, called magnetofection, was initially developed mainly to improve gene transfer in cell cultures, a simpler and ...
arxiv
Performance of AAV8 vectors expressing human factor IX from a hepatic-selective promoter following intravenous injection into rats [PDF]
, 2008 Background: Vectors based on adeno-associated virus-8 (AAV8) have shown efficiency and efficacy for liver-directed gene therapy protocols following intravascular injection, particularly in relation to haemophilia gene therapy. AAV8 has also been proposed
Baker, AH +4 more
core +2 more sources
Network-based Distance Metric with Application to Discover Disease Subtypes in Cancer [PDF]
arXiv, 2017 While we once thought of cancer as single monolithic diseases affecting a specific organ site, we now understand that there are many subtypes of cancer defined by unique patterns of gene mutations. These gene mutational data, which can be more reliably obtained than gene expression data, help to determine how the subtypes develop, evolve, and respond ...
arxiv
The fetal mouse is a sensitive genotoxicity model that exposes lentiviral-associated mutagenesis resulting in liver oncogenesis [PDF]
, 2012 This article is available open access through the publisher’s website at the link below. Copyright @ 2013 The American Society of Gene & Cell Therapy.Genotoxicity models are extremely important to assess retroviral vector biosafety before gene therapy ...
Al-Allaf, F +26 more
core +1 more source
mBio, 2019 Secretion of interleukin-1β (IL-1β) represents a fundamental innate immune response to microbial infection that, at the molecular level, occurs following activation of proteolytic caspases that cleave the immature protein into a secretable form.
Sara Botto +8 more
doaj +1 more source
Adeno‐associated virus serotype 1‐based gene therapy for FTD caused by GRN mutations
Annals of Clinical and Translational Neurology, 2020 Objective Dominant loss‐of‐function mutations in the gene encoding the lysosomal protein, progranulin, cause 5‐10% of frontotemporal dementia cases. As progranulin undergoes secretion and endocytosis, a small number of progranulin‐expressing cells can ...
Christian Hinderer +6 more
doaj +1 more source
, 2015 Cancer treatment has been the major goal of the gene therapy studies over the decades. Although there is no cancer gene therapy drug in the market yet, substantial progress has been made in defining potential targets and in developing viral and nonviral ...
Akbulut, Hakan +2 more
core +2 more sources
ApoE gene therapy: an overview and update [PDF]
, 2005 Atherosclerosis remains the leading cause of death in industrialized societies. Apolipoprotein E (ApoE) is an attractive candidate to treat hypercholesterolemia and coronary heart disease, as it is a circulating protein with pleiotropic ...
Owen, J.S.
core
Response to combination therapy with interferon alfa-2a and ribavirin in chronic hepatitis C according to a TNF-alpha promoter polymorphism [PDF]
, 2003 Background. Tumor necrosis factor-alpha (TNF-alpha) is involved in the pathogenesis of chronic active hepatitis C. Polymorphisms in the promoter region of the TNF-alpha gene can alter the TNF-alpha expression and modify the host immune response.
Folwaczny, Christian +8 more
core +1 more source
AAV gene replacement therapy for treating MPS IIIC: Facilitating bystander effects via EV‐mRNA cargo
Journal of Extracellular VesiclesMPS IIIC is a lysosomal storage disease caused by mutations in heparan‐α‐glucosaminide N‐acetyltransferase (HGSNAT), for which no treatment is available.
Tierra A. Bobo +6 more
doaj +1 more source