Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer
Nature Communications, 2018 Patients with mutations in the ASL gene present with argininosuccinic aciduria characterised by hyperammonaemia and cognitive impairment. Here, the authors show that cerebral disease involves neuronal nitrosative/oxidative stress that is not induced by ...
Julien Baruteau +21 more
doaj +1 more source
Haematologica, 2014 Complement blockade by eculizumab is clinically effective in hemolytic paroxysmal nocturnal hemoglobinuria. However, the response is variable and some patients remain dependent on red blood cell transfusions.
Tommaso Rondelli +14 more
doaj +1 more source
Gene Therapy for Huntington’s Disease: The Final Strategy for a Cure? [PDF]
Journal of Movement Disorders, 2022 Huntington’s disease (HD) has become a target of the first clinical trials for gene therapy among movement disorders with a genetic origin. More than 100 clinical trials regarding HD have been tried, but all failed, although there were some improvements ...
Seulgi Byun, Mijung Lee, Manho Kim
doaj +1 more source
Intratracheal aerosolization of viral vectors to newborn pig airways
BioTechniques, 2020 Gene therapy for airway diseases requires efficient delivery of nucleic acids to the airways. In small animal models, gene delivery reagents are commonly delivered as a bolus dose.
Ashley L Cooney, Patrick L Sinn
doaj +1 more source
Lateral gene transfer between prokaryotes and multicellular eukaryotes: ongoing and significant? [PDF]
, 2009 The expansion of genome sequencing projects has produced accumulating evidence for lateral transfer of genes between prokaryotic and eukaryotic genomes. However, it remains controversial whether these genes are of functional importance in their recipient
Ros Vera ID +5 more
core +1 more source
The Routes of Emergence of Life from LUCA during the RNA and Viral World: A Conspectus
Life, 2015 How did life emerge on Earth? The aim of the Network of Researchers on Horizontal Gene Transfer and the Last Universal Cellular Ancestor (NoR HGT & LUCA) is to understand how the genetics of LUCAs were reorganised prior to the advent of the three ...
Sohan Jheeta
doaj +1 more source
Futuristic Non-antibiotic Therapies to Combat Antibiotic Resistance: A Review
Frontiers in Microbiology, 2021 The looming problem of resistance to antibiotics in microorganisms is a global health concern. The drug-resistant microorganisms originating from anthropogenic sources and commercial livestock farming have posed serious environmental and health ...
Manoj Kumar +9 more
doaj +1 more source
Gene transfer into hepatocytes using asialoglycoprotein receptor mediated endocytosis of DNA complexed with an artificial tetra-antennary galactose ligand [PDF]
, 1992 We have constructed an artificial ligand for the hepatocyte-specific asialoglycoprotein receptor for the purpose of generating a synthetic delivery system for DNA.
Zatloukal, Kurt +9 more
core +1 more source
Journal of Immunology Research, 2015 Following fetal or neonatal gene transfer in mice and other species immune tolerance of the transgenic protein is frequently observed; however the underlying mechanisms remain largely undefined.
Megha S. Nivsarkar +7 more
doaj +1 more source
Binding Partners of Alzheimer's Disease Proteins: Are They Physiologically Relevant?
Neurobiology of Disease, 2000 Protein–protein interactions are a molecular basis for the structural and functional organization within cells. They are mediated by a growing number of protein modules that bind peptide targets.
Geert Van Gassen +2 more
doaj +1 more source