Results 71 to 80 of about 1,824,389 (266)
BMC Genomics, 2020 An amendment to this paper has been published and can be accessed via the original article.
Camilo Breton +4 more
doaj +1 more source
Evolution and Controllability of Cancer Networks: a Boolean Perspective [PDF]
, 2013 Cancer forms a robust system and progresses as stages over time typically with increasing aggressiveness and worsening prognosis. Characterizing these stages and identifying the genes driving transitions between them is critical to understand cancer progression and to develop effective anti-cancer therapies.
arxiv +1 more source
Pupillometric analysis for assessment of gene therapy in Leber Congenital Amaurosis patients [PDF]
, 2012 Background: Objective techniques to assess the amelioration of vision in patients with impaired visual function are needed to standardize efficacy assessment in gene therapy trials for ocular diseases.
Bennett, Jean, M.D. +5 more
core +3 more sources
Gene therapy for arthritis [PDF]
Modern Rheumatology, 2008 Arthritis is among the leading causes of disability in the developed world. There remains no cure for this disease and the current treatments are only modestly effective at slowing the disease's progression and providing symptomatic relief. The clinical effectiveness of current treatment regimens has been limited by short half-lives of the drugs and ...
Raphael Hirsch, Russell S. Traister
openaire +3 more sources
Early induction of cytokine release syndrome by rapidly generated CAR T cells in preclinical models
EMBO Molecular MedicineCytokine release syndrome (CRS) is a significant side-effect of conventional chimeric antigen receptor (CAR) T-cell therapy. To facilitate patient accessibility, short-term (st) CAR T cells, which are administered to patients only 24 h after vector ...
Arezoo Jamali +5 more
doaj +1 more source
Efficient Prodrug Activator Gene Therapy by Retroviral Replicating Vectors Prolongs Survival in an Immune-Competent Intracerebral Glioma Model. [PDF]
, 2020 Prodrug activator gene therapy mediated by murine leukemia virus (MLV)-based retroviral replicating vectors (RRV) was previously shown to be highly effective in killing glioma cells both in culture and in vivo.
Chang, Deching +13 more
core +2 more sources
Oral Diseases, 2013 Applications of gene therapy have been evaluated in virtually every oral tissue, and many of these have proved successful at least in animal models. While gene therapy will not be used routinely in the next decade, practitioners of oral medicine should be aware of the potential of this novel type of treatment that doubtless will benefit many patients ...
openaire +2 more sources
Gene Therapy for Hemophilia [PDF]
Molecular Therapy, 2016 The X-linked bleeding disorder hemophilia causes frequent and exaggerated bleeding that can be life-threatening if untreated. Conventional therapy requires frequent intravenous infusions of the missing coagulation protein (factor VIII [FVIII] for hemophilia A and factor IX [FIX] for hemophilia B).
Nienhuis, AW, Nathwani, AC, Davidoff, AM
openaire +4 more sources
Incorporation of aptamers in the terminal loop of shRNAs yields an effective and novel combinatorial targeting strategy. [PDF]
, 2017 Gene therapy by engineering patient's own blood cells to confer HIV resistance can potentially lead to a functional cure for AIDS. Toward this goal, we have previously developed an anti-HIV lentivirus vector that deploys a combination of shRNA, ribozyme ...
Castanotto, Daniela +4 more
core +1 more source
FV Vectors as Alternative Gene Vehicles for Gene Transfer in HSCs
Viruses, 2020 Hematopoietic Stem Cells (HSCs) are a unique population of cells, capable of reconstituting the blood system of an organism through orchestrated self-renewal and differentiation.
Emmanouil Simantirakis +2 more
doaj +1 more source