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Lista de obras de Alberto Auricchio

A Comprehensive Map of CNS Transduction by Eight Recombinant Adeno-associated Virus Serotypes Upon Cerebrospinal Fluid Administration in Pigs

artículo científico publicado en 2015

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

artículo científico publicado en 2001

AAV-mediated gene replacement, either alone or in combination with physical and pharmacological agents, results in partial and transient protection from photoreceptor degeneration associated with betaPDE deficiency

artículo científico publicado en 2011

AAV-mediated gene supply for treatment of degenerative and neovascular retinal diseases.

artículo científico publicado en 2010

AAV-mediated gene transfer for retinal diseases

artículo científico publicado en 2006

AAV-mediated liver-specific MPV17 expression restores mtDNA levels and prevents diet-induced liver failure

artículo científico publicado en 2014

AAV-mediated tyrosinase gene transfer restores melanogenesis and retinal function in a model of oculo-cutaneous albinism type I (OCA1).

artículo científico publicado en 2009

AAV2 gene therapy readministration in three adults with congenital blindness.

artículo científico publicado en 2012

AAV2/8 vectors purified from culture medium with a simple and rapid protocol transduce murine liver, muscle, and retina efficiently

artículo científico publicado en 2013

AP20187-mediated activation of a chimeric insulin receptor results in insulin-like actions in skeletal muscle and liver of diabetic mice.

artículo científico publicado en 2007

Aberrant splicing in the ocular albinism type 1 gene (OA1/GPR143) is corrected in vitro by morpholino antisense oligonucleotides.

artículo científico publicado en 2006

Adeno-associated viral vectors for retinal gene transfer and treatment of retinal diseases

artículo científico publicado en 2005

Adeno-associated viral vectors for retinal gene transfer.

artículo científico publicado en 2003

Adenoviral vector-mediated insulin gene transfer in the mouse pancreas corrects streptozotocin-induced hyperglycemia.

artículo científico publicado en 2001

Advances in AAV-mediated gene transfer for the treatment of inherited disorders.

artículo científico publicado en 2004

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

artículo científico publicado en 2009

Amelioration of both functional and morphological abnormalities in the retina of a mouse model of ocular albinism following AAV-mediated gene transfer

artículo científico publicado en 2005

An in vivo doxycycline-controlled expression system for functional studies of the retina

artículo científico publicado en 2003

An innovative strategy for the molecular diagnosis of Usher syndrome identifies causal biallelic mutations in 93% of European patients

artículo científico publicado en 2016

Autonomic dysfunction in children with Hirschsprung's disease

artículo científico publicado en 1999

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

scientific journal article

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

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

artículo científico publicado en 2007

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

artículo científico publicado en 2006

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

artículo científico publicado en 2010

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

artículo científico publicado en 2007

CoCas9 is a compact nuclease from the human microbiome for efficient and precise genome editing

artículo científico publicado en 2024

Combined rod and cone transduction by adeno-associated virus 2/8.

scientific article published on 30 October 2013

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

artículo científico publicado en 2006

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

artículo científico publicado en 2012

Cross-talk between androgen receptor/filamin A and TrkA regulates neurite outgrowth in PC12 cells

artículo científico publicado en 2015

Delivery of adeno-associated virus vectors to the fetal retina: impact of viral capsid proteins on retinal neuronal progenitor transduction

artículo científico publicado en 2003

Disease rescue and increased lifespan in a model of cardiomyopathy and muscular dystrophy by combined AAV treatments

artículo científico publicado en 2009

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

artículo científico publicado en 1999

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

artículo científico publicado en 2012

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

artículo científico publicado en 2013

Efficacy of a combined intracerebral and systemic gene delivery approach for the treatment of a severe lysosomal storage disorder

artículo científico publicado en 2011

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

artículo científico publicado en 2014

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

artículo científico publicado en 2003

En Face Spectral-Domain Optical Coherence Tomography for the Monitoring of Lesion Area Progression in Stargardt Disease

artículo científico publicado en 2016

Epidermal growth factor induces protein tyrosine phosphorylation and association of p190 with ras-GTP-ase activating protein in Caco-2 cells

artículo científico publicado en 1994

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

artículo científico publicado en 2011

Fighting Blindness with Adeno-Associated Virus Serotype 8 ⬇️

artículo científico publicado el 1 de octubre de 2011

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

artículo científico publicado en 2007

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

artículo científico publicado en 2007

Gene Therapy of ABCA4-Associated Diseases

artículo científico publicado en 2015

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

artículo científico publicado en 2009

Gene therapy for mucopolysaccharidosis type VI is effective in cats without pre-existing immunity to AAV8.

artículo científico publicado en 2013

Gene therapy of inherited retinal degenerations: prospects and challenges

artículo científico publicado en 2015

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

artículo científico publicado en 2012

Gene therapy using a liver-targeted AAV vector restores nucleoside and nucleotide homeostasis in a murine model of MNGIE.

artículo científico publicado en 2014

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

artículo científico publicado en 2015

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

artículo científico publicado en 2001

Impact of age at administration, lysosomal storage, and transgene regulatory elements on AAV2/8-mediated rat liver transduction

artículo científico publicado en 2012

Improved dual AAV vectors with reduced expression of truncated proteins are safe and effective in the retina of a mouse model of Stargardt disease

artículo científico publicado en 2015

In vivo quantitative noninvasive imaging of gene transfer by single-photon emission computerized tomography.

artículo científico publicado en 2003

Inhibition of ocular neovascularization by hedgehog blockade.

artículo científico publicado en 2005

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

scientific article published on 01 October 2002

Intein-mediated protein trans-splicing expands adeno-associated virus transfer capacity in the retina

scientific article published on 01 May 2019

Intramuscular viral delivery of paraplegin rescues peripheral axonopathy in a model of hereditary spastic paraplegia

artículo científico publicado en 2005

Intramuscular viral delivery of paraplegin rescues peripheral axonopathy in amodel of hereditary spastic paraplegia.

artículo científico publicado en 2014

Isolation and evaluation of novel adeno-associated virus sequences from porcine tissues.

artículo científico publicado en 2009

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

artículo científico publicado en 2001

Large gene delivery to the retina with AAV vectors: are we there yet?

artículo científico publicado en 2020

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

artículo científico publicado en 2010

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

artículo científico publicado en 2008

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

artículo científico publicado en 2005

Low-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease

artículo científico publicado en 2016

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

artículo científico publicado en 2014

MicroRNA-restricted transgene expression in the retina

artículo científico publicado en 2011

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

artículo científico publicado en 2010

Myosin7a deficiency results in reduced retinal activity which is improved by gene therapy

artículo científico publicado en 2013

Natural antisense transcripts associated with genes involved in eye development

artículo científico publicado en 2005

Non-erythropoietic erythropoietin derivatives protect from light-induced and genetic photoreceptor degeneration.

artículo científico publicado en 2011

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

artículo científico publicado en 2002

Noninvasive repetitive imaging of somatostatin receptor 2 gene transfer with positron emission tomography.

artículo científico publicado en 2011

Novel adeno-associated viral vectors for retinal gene therapy ⬇️

artículo científico publicado el 13 de octubre de 2011

Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors

artículo científico publicado en 2007

Novel adeno-associated viruses derived from pig tissues transduce most major organs in mice

artículo científico publicado en 2014

Ocular gene therapy: current progress and future prospects

artículo científico publicado en 2008

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

artículo científico publicado en 2004

Pharmacological read-through of nonsense ARSB mutations as a potential therapeutic approach for mucopolysaccharidosis VI

artículo científico publicado en 2012

Photoreceptor degeneration in mice: adeno-associated viral vector-mediated delivery of erythropoietin.

artículo científico publicado en 2013

Preferential silencing of a common dominant rhodopsin mutation does not inhibit retinal degeneration in a transgenic model

article

Prevalence of anti-adeno-associated virus serotype 8 neutralizing antibodies and arylsulfatase B cross-reactive immunologic material in mucopolysaccharidosis VI patient candidates for a gene therapy trial.

artículo científico publicado en 2015

Proliferation of Multiple Cell Types in the Skeletal Muscle Tissue Elicited by Acute p21 Suppression

artículo científico publicado en 2015

Pseudotyped AAV vectors for constitutive and regulated gene expression in the eye.

artículo científico publicado en 2003

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

artículo científico publicado en 2007

Recombinant vectors based on porcine adeno-associated viral serotypes transduce the murine and pig retina

artículo científico publicado en 2013

Retinal transduction profiles by high-capacity viral vectors

artículo científico publicado en 2014

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

artículo científico publicado en 2007

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.

artículo científico publicado en 2016

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

artículo científico publicado en 2008

Sensory-motor behavioral characterization of an animal model of Maroteaux-Lamy syndrome (or Mucopolysaccharidosis VI).

artículo científico publicado en 2014

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

artículo científico publicado en 2008

Similar therapeutic efficacy between a single administration of gene therapy and multiple administrations of recombinant enzyme in a mouse model of lysosomal storage disease

artículo científico publicado en 2014

Single delivery of an adeno-associated viral construct to transfer the CASQ2 gene to knock-in mice affected by catecholaminergic polymorphic ventricular tachycardia is able to cure the disease from birth to advanced age.

artículo científico publicado en 2014

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

artículo científico publicado en 2004

The future looks brighter after 25 years of retinal gene therapy

artículo científico

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

artículo científico publicado en 2011

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

artículo científico publicado en 2011

The longevity-associated variant of BPIFB4 improves a CXCR4-mediated striatum-microglia crosstalk preventing disease progression in a mouse model of Huntington's disease

scientific article published on 18 July 2020

The microphthalmia transcription factor (Mitf) controls expression of the ocular albinism type 1 gene: link between melanin synthesis and melanosome biogenesis

artículo científico publicado en 2004

Therapeutic homology-independent targeted integration in retina and liver

scientific article published on 12 April 2022

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

artículo científico publicado en 2013

Use of a lower dosage liver-detargeted AAV vector to prevent hamster muscular dystrophy

artículo científico publicado en 2013

Vector platforms for gene therapy of inherited retinopathies.

artículo científico publicado en 2014

Versatility of AAV vectors for retinal gene transfer

artículo científico publicado en 2007

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

artículo científico publicado en 2012

Worsening of cardiomyopathy using deflazacort in an animal model rescued by gene therapy

artículo científico publicado en 2011

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

artículo científico publicado en 2011

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