Disease Models & Mechanisms, 2016 Mucopolysaccharidosis type IIIC (MPSIIIC) is a severe lysosomal storage disease caused by deficiency in activity of the transmembrane enzyme heparan-α-glucosaminide N-acetyltransferase (HGSNAT) that catalyses the N-acetylation of α-glucosamine residues ...
Sara Marcó +14 more
doaj +1 more source
Metastatic melanoma-A review of current and future perspective [PDF]
arXiv, 2020 Metastatic Melanoma, the fifth most common cancer in the western countries and the most common malignancy diagnosed in United States present itself as the most lethal treatment resistant cancer worldwide. In addition to the reactive oxygen species(ROS), mutations in the genes encoding receptors and non-receptor tyrosine/serene/threonine protein kinases
arxiv
Assembly and functional analysis of an S/MAR based episome with the cystic fibrosis transmembrane conductance regulator gene [PDF]
, 2018 Improving the efficacy of gene therapy vectors is still an important goal toward the development of safe and efficient gene therapy treatments. S/MAR (scaffold/matrix attached region)-based vectors are maintained extra-chromosomally in numerous cell ...
Ascenzioni, Fiorentina +10 more
core +2 more sources
Gene therapy for epilepsy [PDF]
Epilepsy & Behavior, 2014 Gene therapy may represent an effective alternative to standard pharmacological approaches for certain forms of epilepsy. Currently, the best candidates for this therapeutic approach appear to be epilepsies characterized by a focal lesion. Gene therapy has been attempted to produce antiepileptogenic (prevention of development of epilepsy in subject at ...
openaire +5 more sources
Gene Therapy for Human Genetic Disease? [PDF]
, 1972 Elizabeth F. Neufeld, Charles C. Sweeley
openalex +1 more source
Surface immobilization of hexa-histidine-tagged adeno-associated viral vectors for localized gene delivery. [PDF]
, 2010 Adeno-associated viral (AAV) vectors, which are undergoing broad exploration in clinical trials, have significant promise for therapeutic gene delivery because of their safety and delivery efficiency.
Bethi, SR +5 more
core +2 more sources
Universal Method for the Purification of Recombinant AAV Vectors of Differing Serotypes
Molecular Therapy: Methods & Clinical Development, 2018 The generation of clinical good manufacturing practices (GMP)-grade adeno-associated virus (AAV) vectors requires purification strategies that support the generation of vectors of high purity, and that exhibit a good safety and efficacy profile. To date,
Shelley A. Nass +9 more
doaj
Molecular Therapy: Methods & Clinical Development, 2023 Limb-girdle muscular dystrophy (LGMD) type 2C/R5 results from mutations in the γ-sarcoglycan (SGCG) gene and is characterized by muscle weakness and progressive wasting.
Young-Eun Seo +11 more
doaj
Implantation of fibroblasts transfected with human granulocyte colony- stimulating factor cDNA into mice as a model of cytokine-supplement gene therapy [PDF]
, 1989 Kenzaburo Tani +9 more
openalex +1 more source
Impact of polyplex micelles installed with cyclic RGD peptide as ligand on gene delivery to vascular lesions [PDF]
, 2010 Gene therapy is expected to open a new strategy for the treatment of refractory vascular diseases, so the development of appropriate gene vectors for vascular lesions is needed. To realize this requirement with a non-viral approach, cyclo(RGDfK) peptide (
Hideo Kagaya +7 more
core +1 more source