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List of works by Alberto Auricchio

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

scientific article published on 7 December 2015

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

scientific article

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

scientific article published on 29 July 2011

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

scientific article published on October 2010

AAV-mediated gene transfer for retinal diseases

scientific article published on December 2006

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

scientific article

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

scientific article published on 12 May 2009

AAV2 gene therapy readministration in three adults with congenital blindness.

scientific article

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

scientific article published on 11 October 2013

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

scientific article

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

scientific article

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

scientific article

Adeno-associated viral vectors for retinal gene transfer.

scientific article published on November 2003

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

scientific article

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

scientific article published on April 2004

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

scientific article

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

scientific article published in October 2005

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

scientific article

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

scientific article published on 27 July 2016

Autonomic dysfunction in children with Hirschsprung's disease

scientific article published in 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.

scientific article published on 23 October 2007

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

scholarly article

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

scientific article

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

scientific article published on September 2007

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

scientific article published in 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

scientific article published on 27 February 2006

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

scientific article

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

scientific article

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

scientific article

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

scientific article (publication date: 2009)

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

scientific article published in April 1999

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

scientific article

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

scientific article published on 15 December 2013

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

scientific article

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

scientific article published on 27 February 2014

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

scientific article

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

scientific article published on July 2016

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

scientific article published on 01 October 1994

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

Fighting Blindness with Adeno-Associated Virus Serotype 8 ⬇️

scientific article published on October 1, 2011

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

scientific article published on 26 February 2007

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

Gene Therapy of ABCA4-Associated Diseases

scientific article published on 8 January 2015

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

scientific article

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

scientific article published on 30 January 2013

Gene therapy of inherited retinal degenerations: prospects and challenges

scientific article

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

scientific article

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

scientific article published on 22 January 2014

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

scientific article

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

scientific article published on July 2001

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

scientific article (publication date: 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

scientific article

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

scientific article published in February 2003

Inhibition of ocular neovascularization by hedgehog blockade.

scientific article

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

scientific article

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

scientific article

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

scientific article published on 23 July 2009

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

scientific article

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

scientific article published on 13 July 2020

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

scientific article

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

scientific article published in June 2008

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

scientific article published in February 2005

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

scientific article published on 23 September 2016

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

scientific article

MicroRNA-restricted transgene expression in the retina

scientific article

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

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

scientific article

Natural antisense transcripts associated with genes involved in eye development

scientific article

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

scientific article published on 19 March 2011

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

scientific article

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

scientific article published on 11 January 2011

Novel adeno-associated viral vectors for retinal gene therapy ⬇️

scientific article published on October 13, 2011

Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors

scientific article

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

scientific article

Ocular gene therapy: current progress and future prospects

scientific article published on 25 December 2008

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

scientific article published on January 2004

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

scientific article published on 13 September 2012

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

scientific article published in January 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.

scientific article

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

scientific article published on 11 February 2015

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

scientific article

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

scientific article published on 23 May 2007

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

scientific article

Retinal transduction profiles by high-capacity viral vectors

scientific article published on 3 July 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

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

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

scientific article

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

scientific article

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

scientific article published on May 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

scientific article

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.

scientific article published on 2 June 2014

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

scientific article published in November 2004

The future looks brighter after 25 years of retinal gene therapy

scientific article published on 21 August 2017

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

scientific article

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

scientific article published in July 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

scientific article

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

scientific article published on 6 March 2013

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

scientific article

Vector platforms for gene therapy of inherited retinopathies.

scientific article

Versatility of AAV vectors for retinal gene transfer

scientific article published on 17 October 2007

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

scientific article

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

scientific article

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

scientific article

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