List of works - Alberto Auricchio

Safety and efficacy of gene transfer for Leber's congenital amaurosis

scientific article

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

scientific article

Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration

scientific article

AAV2 gene therapy readministration in three adults with congenital blindness.

scientific article

Isolation of highly infectious and pure adeno-associated virus type 2 vectors with a single-step gravity-flow column.

scientific article

Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2

scientific article published on 6 March 2013

Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice.

scientific article published on May 2008

Hybrid vectors based on adeno-associated virus serotypes 2 and 5 for muscle-directed gene transfer

scientific article published on July 2001

MicroRNAs Involved in Molecular Circuitries Relevant for the Duchenne Muscular Dystrophy Pathogenesis Are Controlled by the Dystrophin/nNOS Pathway

scientific article published on 19 August 2010

Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors

scientific article

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial.

scientific article published on 30 June 2016

Axonal degeneration in paraplegin-deficient mice is associated with abnormal mitochondria and impairment of axonal transport

scientific journal article

RNA interference-mediated suppression and replacement of human rhodopsin in vivo

scientific article published on 23 May 2007

Axonal degeneration in paraplegin-deficient mice is associated with abnormal mitochondria and impairment of axonal transport

Noninvasive gene transfer to the lung for systemic delivery of therapeutic proteins.

scientific article

PEGylation of a vesicular stomatitis virus G pseudotyped lentivirus vector prevents inactivation in serum

scientific article published on January 2004

Effective delivery of large genes to the retina by dual AAV vectors

scientific article published on 15 December 2013

Clinical and molecular genetics of Leber's congenital amaurosis: a multicenter study of Italian patients

scientific article published on September 2007

The human visual cortex responds to gene therapy-mediated recovery of retinal function

scientific article

Novel adeno-associated viral vectors for retinal gene therapy

scientific article published on October 13, 2011

Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model.

scholarly article

Natural antisense transcripts associated with genes involved in eye development

scientific article

Versatility of AAV vectors for retinal gene transfer

scientific article published on 17 October 2007

Correction of Hunter syndrome in the MPSII mouse model by AAV2/8-mediated gene delivery

scientific article published on 27 February 2006

Systemic but not intraocular Epo gene transfer protects the retina from light-and genetic-induced degeneration

scientific article published in November 2004

Functional correction of CNS lesions in an MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes

scientific article published on 27 August 2007

Efficient trans-splicing in the retina expands the utility of adeno-associated virus as a vector for gene therapy.

scientific article

Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material

scientific article

Vector platforms for gene therapy of inherited retinopathies.

scientific article

Efficient gene delivery to the cone-enriched pig retina by dual AAV vectors

scientific article published on 27 February 2014

Double heterozygosity for a RET substitution interfering with splicing and an EDNRB missense mutation in Hirschsprung disease

scientific article published in April 1999

Biochemical, pathological, and skeletal improvement of mucopolysaccharidosis VI after gene transfer to liver but not to muscle.

scientific article published on 23 October 2007

A single-step affinity column for purification of serotype-5 based adeno-associated viral vectors.

scientific article

Long-term benefit of adeno-associated virus/antisense-mediated exon skipping in dystrophic mice.

scientific article published in June 2008

Inhibition of retinal neovascularization by intraocular viral-mediated delivery of anti-angiogenic agents

scientific article published on 01 October 2002

Filamin A is mutated in X-linked chronic idiopathic intestinal pseudo-obstruction with central nervous system involvement

scientific article published on 26 February 2007

Zinc-finger-based transcriptional repression of rhodopsin in a model of dominant retinitis pigmentosa

scientific article

MicroRNA-restricted transgene expression in the retina

scientific article

SEPN1, an endoplasmic reticulum-localized selenoprotein linked to skeletal muscle pathology, counteracts hyperoxidation by means of redox-regulating SERCA2 pump activity

scientific journal article

SUMF1 enhances sulfatase activities in vivo in five sulfatase deficiencies

scientific article

Long-term amelioration of feline Mucopolysaccharidosis VI after AAV-mediated liver gene transfer

scientific article

Ocular gene therapy: current progress and future prospects

scientific article published on 25 December 2008

Effective AAV-mediated gene therapy in a mouse model of ethylmalonic encephalopathy

scientific article

Correlation between photoreceptor layer integrity and visual function in patients with Stargardt disease: implications for gene therapy

scientific article

Long-term inducible gene expression in the eye via adeno-associated virus gene transfer in nonhuman primates.

scientific article published in February 2005

Manufacturing and characterization of a recombinant adeno-associated virus type 8 reference standard material.

scientific article

Genetic Analysis Reveals a Longevity-Associated Protein Modulating Endothelial Function and Angiogenesis

scientific article

Viral gene transfer rescues arrhythmogenic phenotype and ultrastructural abnormalities in adult calsequestrin-null mice with inherited arrhythmias

scientific article

Evaluation of Italian patients with leber congenital amaurosis due to AIPL1 mutations highlights the potential applicability of gene therapy

scientific article published on 29 July 2011

Gene therapy of inherited retinopathies: a long and successful road from viral vectors to patients

scientific article

List of works by Alberto Auricchio

Safety and efficacy of gene transfer for Leber's congenital amaurosis

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration

AAV2 gene therapy readministration in three adults with congenital blindness.

Isolation of highly infectious and pure adeno-associated virus type 2 vectors with a single-step gravity-flow column.

Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2

Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice.

Hybrid vectors based on adeno-associated virus serotypes 2 and 5 for muscle-directed gene transfer

MicroRNAs Involved in Molecular Circuitries Relevant for the Duchenne Muscular Dystrophy Pathogenesis Are Controlled by the Dystrophin/nNOS Pathway

Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial.

Axonal degeneration in paraplegin-deficient mice is associated with abnormal mitochondria and impairment of axonal transport

RNA interference-mediated suppression and replacement of human rhodopsin in vivo

Axonal degeneration in paraplegin-deficient mice is associated with abnormal mitochondria and impairment of axonal transport

Noninvasive gene transfer to the lung for systemic delivery of therapeutic proteins.

PEGylation of a vesicular stomatitis virus G pseudotyped lentivirus vector prevents inactivation in serum

Effective delivery of large genes to the retina by dual AAV vectors

Clinical and molecular genetics of Leber's congenital amaurosis: a multicenter study of Italian patients

The human visual cortex responds to gene therapy-mediated recovery of retinal function

Novel adeno-associated viral vectors for retinal gene therapy

Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model.

Natural antisense transcripts associated with genes involved in eye development

Versatility of AAV vectors for retinal gene transfer

Correction of Hunter syndrome in the MPSII mouse model by AAV2/8-mediated gene delivery

Systemic but not intraocular Epo gene transfer protects the retina from light-and genetic-induced degeneration

Functional correction of CNS lesions in an MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes

Efficient trans-splicing in the retina expands the utility of adeno-associated virus as a vector for gene therapy.

Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material

Vector platforms for gene therapy of inherited retinopathies.

Efficient gene delivery to the cone-enriched pig retina by dual AAV vectors

Double heterozygosity for a RET substitution interfering with splicing and an EDNRB missense mutation in Hirschsprung disease

Biochemical, pathological, and skeletal improvement of mucopolysaccharidosis VI after gene transfer to liver but not to muscle.

A single-step affinity column for purification of serotype-5 based adeno-associated viral vectors.

Long-term benefit of adeno-associated virus/antisense-mediated exon skipping in dystrophic mice.

Inhibition of retinal neovascularization by intraocular viral-mediated delivery of anti-angiogenic agents

Filamin A is mutated in X-linked chronic idiopathic intestinal pseudo-obstruction with central nervous system involvement

Zinc-finger-based transcriptional repression of rhodopsin in a model of dominant retinitis pigmentosa

MicroRNA-restricted transgene expression in the retina

SEPN1, an endoplasmic reticulum-localized selenoprotein linked to skeletal muscle pathology, counteracts hyperoxidation by means of redox-regulating SERCA2 pump activity

SUMF1 enhances sulfatase activities in vivo in five sulfatase deficiencies

Long-term amelioration of feline Mucopolysaccharidosis VI after AAV-mediated liver gene transfer

Ocular gene therapy: current progress and future prospects

Effective AAV-mediated gene therapy in a mouse model of ethylmalonic encephalopathy

Correlation between photoreceptor layer integrity and visual function in patients with Stargardt disease: implications for gene therapy

Long-term inducible gene expression in the eye via adeno-associated virus gene transfer in nonhuman primates.

Manufacturing and characterization of a recombinant adeno-associated virus type 8 reference standard material.

Genetic Analysis Reveals a Longevity-Associated Protein Modulating Endothelial Function and Angiogenesis

Viral gene transfer rescues arrhythmogenic phenotype and ultrastructural abnormalities in adult calsequestrin-null mice with inherited arrhythmias

Evaluation of Italian patients with leber congenital amaurosis due to AIPL1 mutations highlights the potential applicability of gene therapy

Gene therapy of inherited retinopathies: a long and successful road from viral vectors to patients