about
CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome)In vivo genome editing restores haemostasis in a mouse model of haemophiliaTreatment of diabetes and long-term survival after insulin and glucokinase gene therapy.Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapyAAV-mediated pancreatic overexpression of Igf1 counteracts progression to autoimmune diabetes in mice.Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B.Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs.Insulin-like growth factor I (IGF-I)-induced chronic gliosis and retinal stress lead to neurodegeneration in a mouse model of retinopathyRobust ZFN-mediated genome editing in adult hemophilic mice.Progressive neurologic and somatic disease in a novel mouse model of human mucopolysaccharidosis type IIIC.Proliferative retinopathies: animal models and therapeutic opportunities.Insulin-like Growth Factor 2 Overexpression Induces β-Cell Dysfunction and Increases Beta-cell Susceptibility to Damage.Inhibition of hepatitis C virus replication using adeno-associated virus vector delivery of an exogenous anti-hepatitis C virus microRNA cluster.Long-Term Efficacy and Safety of Insulin and Glucokinase Gene Therapy for Diabetes: 8-Year Follow-Up in Dogs.Disease correction by AAV-mediated gene therapy in a new mouse model of mucopolysaccharidosis type IIID.Increased ocular levels of IGF-1 in transgenic mice lead to diabetes-like eye disease.Modulation of CD8+ T cell responses to AAV vectors with IgG-derived MHC class II epitopesToward a gene therapy for neurological and somatic MPSIIIABiochemical, histological and functional correction of mucopolysaccharidosis type IIIB by intra-cerebrospinal fluid gene therapy.In vivo gene transfer to pancreatic beta cells by systemic delivery of adenoviral vectors.IGF-I mediates regeneration of endocrine pancreas by increasing beta cell replication through cell cycle protein modulation in mice.
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description
investigador
@es
researcher
@en
wetenschapper
@nl
name
Virginia Haurigot
@en
Virginia Haurigot
@nl
type
label
Virginia Haurigot
@en
Virginia Haurigot
@nl
prefLabel
Virginia Haurigot
@en
Virginia Haurigot
@nl
P108
P31
P496
0000-0002-9772-2565