List of works - Anthony Donsante

AAV vector integration sites in mouse hepatocellular carcinoma.

scientific article published in July 2007

ATP7A gene addition to the choroid plexus results in long-term rescue of the lethal copper transport defect in a Menkes disease mouse model

scientific article published on 30 August 2011

Altered intracellular localization and valosin-containing protein (p97 VCP) interaction underlie ATP7A-related distal motor neuropathy

scientific article published on 30 December 2011

Clinical response to persistent, low-level beta-glucuronidase expression in the murine model of mucopolysaccharidosis type VII.

scientific article published on 16 February 2007

Controlling the Release of Neurotrophin-3 and Chondroitinase ABC Enhances the Efficacy of Nerve Guidance Conduits

scientific article published on 16 June 2020

Differences in ATP7A gene expression underlie intrafamilial variability in Menkes disease/occipital horn syndrome

scientific article published on 11 May 2007

Fetal Brain-directed AAV Gene Therapy Results in Rapid, Robust, and Persistent Transduction of Mouse Choroid Plexus Epithelia

scientific article published on 25 June 2013

Gene Therapy for the Treatment of Neurological Disorders: Amyotrophic Lateral Sclerosis.

scientific article

Gene, Stem Cell, and Alternative Therapies for SCA 1

scientific article

Increased frequency of congenital heart defects in Menkes disease

scientific article

Intracerebroventricular delivery of self-complementary adeno-associated virus serotype 9 to the adult rat brain.

scientific article published on 29 January 2016

L-threo-dihydroxyphenylserine corrects neurochemical abnormalities in a Menkes disease mouse model

scientific article published on 7 December 2012

Molecular and biochemical characterization of a unique mutation in CCS, the human copper chaperone to superoxide dismutase.

scientific article

Molecular correlates of epilepsy in early diagnosed and treated Menkes disease

scientific article

Neonatal diagnosis and treatment of Menkes disease

scientific article

Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors.

scientific article published in September 2001

Prenatal diagnostic conundrum involving a novel ATP7A duplication

scientific article

Progress in gene and cell therapies for the neuronal ceroid lipofuscinoses

article

Somatic mosaicism in Menkes disease suggests choroid plexus‐mediated copper transport to the developing brain ⬇️

scientific article published on October 1, 2010

Translational read-through of a nonsense mutation in ATP7A impacts treatment outcome in Menkes disease.

scientific article

List of works by Anthony Donsante

AAV vector integration sites in mouse hepatocellular carcinoma.

ATP7A gene addition to the choroid plexus results in long-term rescue of the lethal copper transport defect in a Menkes disease mouse model

Altered intracellular localization and valosin-containing protein (p97 VCP) interaction underlie ATP7A-related distal motor neuropathy

Clinical response to persistent, low-level beta-glucuronidase expression in the murine model of mucopolysaccharidosis type VII.

Controlling the Release of Neurotrophin-3 and Chondroitinase ABC Enhances the Efficacy of Nerve Guidance Conduits

Differences in ATP7A gene expression underlie intrafamilial variability in Menkes disease/occipital horn syndrome

Fetal Brain-directed AAV Gene Therapy Results in Rapid, Robust, and Persistent Transduction of Mouse Choroid Plexus Epithelia

Gene Therapy for the Treatment of Neurological Disorders: Amyotrophic Lateral Sclerosis.

Gene, Stem Cell, and Alternative Therapies for SCA 1

Increased frequency of congenital heart defects in Menkes disease

Intracerebroventricular delivery of self-complementary adeno-associated virus serotype 9 to the adult rat brain.

L-threo-dihydroxyphenylserine corrects neurochemical abnormalities in a Menkes disease mouse model

Molecular and biochemical characterization of a unique mutation in CCS, the human copper chaperone to superoxide dismutase.

Molecular correlates of epilepsy in early diagnosed and treated Menkes disease

Neonatal diagnosis and treatment of Menkes disease

Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors.

Prenatal diagnostic conundrum involving a novel ATP7A duplication

Progress in gene and cell therapies for the neuronal ceroid lipofuscinoses

Somatic mosaicism in Menkes disease suggests choroid plexus‐mediated copper transport to the developing brain ⬇️

Translational read-through of a nonsense mutation in ATP7A impacts treatment outcome in Menkes disease.