Lista de obras -

AAV-mediated pancreatic overexpression of Igf1 counteracts progression to autoimmune diabetes in mice.

artículo científico publicado en 2017

Biochemical, histological and functional correction of mucopolysaccharidosis type IIIB by intra-cerebrospinal fluid gene therapy

artículo científico publicado en 2014

CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome)

artículo científico publicado en 2016

Disease correction by AAV-mediated gene therapy in a new mouse model of mucopolysaccharidosis type IIID.

artículo científico publicado en 2017

IGF-I mediates regeneration of endocrine pancreas by increasing beta cell replication through cell cycle protein modulation in mice.

artículo científico publicado en 2008

In vivo gene transfer to pancreatic beta cells by systemic delivery of adenoviral vectors.

artículo científico publicado en 2004

In vivo genome editing restores haemostasis in a mouse model of haemophilia

artículo científico publicado en 2011

Increased ocular levels of IGF-1 in transgenic mice lead to diabetes-like eye disease.

artículo científico publicado en 2004

Inhibition of hepatitis C virus replication using adeno-associated virus vector delivery of an exogenous anti-hepatitis C virus microRNA cluster.

artículo científico publicado en 2010

Insulin-like Growth Factor 2 Overexpression Induces β-Cell Dysfunction and Increases Beta-cell Susceptibility to Damage

artículo científico publicado en 2015

Insulin-like growth factor I (IGF-I)-induced chronic gliosis and retinal stress lead to neurodegeneration in a mouse model of retinopathy

artículo científico publicado en 2013

Long-Term Efficacy and Safety of Insulin and Glucokinase Gene Therapy for Diabetes: 8-Year Follow-Up in Dogs

artículo científico publicado en 2017

Long-Term Retinal PEDF Overexpression Prevents Neovascularization in a Murine Adult Model of Retinopathy ⬇️

artículo científico publicado el 20 de julio de 2012

Modulation of CD8+ T cell responses to AAV vectors with IgG-derived MHC class II epitopes

artículo científico publicado en 2013

Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B.

artículo científico publicado en 2010

Progressive neurologic and somatic disease in a novel mouse model of human mucopolysaccharidosis type IIIC.

artículo científico publicado en 2016

Proliferative retinopathies: animal models and therapeutic opportunities

artículo científico publicado en 2015

Robust ZFN-mediated genome editing in adult hemophilic mice.

artículo científico publicado en 2013

Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs.

artículo científico publicado en 2010

Toward a gene therapy for neurological and somatic MPSIIIA

artículo científico publicado en 2013

Treatment of diabetes and long-term survival after insulin and glucokinase gene therapy.

artículo científico publicado en 2013

Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy

artículo científico publicado en 2013

Lista de obras de

AAV-mediated pancreatic overexpression of Igf1 counteracts progression to autoimmune diabetes in mice.

Biochemical, histological and functional correction of mucopolysaccharidosis type IIIB by intra-cerebrospinal fluid gene therapy

CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome)

Disease correction by AAV-mediated gene therapy in a new mouse model of mucopolysaccharidosis type IIID.

IGF-I mediates regeneration of endocrine pancreas by increasing beta cell replication through cell cycle protein modulation in mice.

In vivo gene transfer to pancreatic beta cells by systemic delivery of adenoviral vectors.

In vivo genome editing restores haemostasis in a mouse model of haemophilia

Increased ocular levels of IGF-1 in transgenic mice lead to diabetes-like eye disease.

Inhibition of hepatitis C virus replication using adeno-associated virus vector delivery of an exogenous anti-hepatitis C virus microRNA cluster.

Insulin-like Growth Factor 2 Overexpression Induces β-Cell Dysfunction and Increases Beta-cell Susceptibility to Damage

Insulin-like growth factor I (IGF-I)-induced chronic gliosis and retinal stress lead to neurodegeneration in a mouse model of retinopathy

Long-Term Efficacy and Safety of Insulin and Glucokinase Gene Therapy for Diabetes: 8-Year Follow-Up in Dogs

Long-Term Retinal PEDF Overexpression Prevents Neovascularization in a Murine Adult Model of Retinopathy ⬇️

Modulation of CD8+ T cell responses to AAV vectors with IgG-derived MHC class II epitopes

Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B.

Progressive neurologic and somatic disease in a novel mouse model of human mucopolysaccharidosis type IIIC.

Proliferative retinopathies: animal models and therapeutic opportunities

Robust ZFN-mediated genome editing in adult hemophilic mice.

Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs.

Toward a gene therapy for neurological and somatic MPSIIIA

Treatment of diabetes and long-term survival after insulin and glucokinase gene therapy.

Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy