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List of works by Alberto Auricchio

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

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

scientific article
Inhibition of ocular neovascularization by hedgehog blockade.

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

scientific article
AAV-mediated gene transfer for retinal diseases

scientific article published on December 2006
Isolation and evaluation of novel adeno-associated virus sequences from porcine tissues.

scientific article published on 23 July 2009
Pseudotyped AAV vectors for constitutive and regulated gene expression in the eye.

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

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

scientific article
Adeno-associated viral vectors for retinal gene transfer.

scientific article published on November 2003
Preferential silencing of a common dominant rhodopsin mutation does not inhibit retinal degeneration in a transgenic model

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

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

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

scientific article (publication date: 2009)
Pharmacological read-through of nonsense ARSB mutations as a potential therapeutic approach for mucopolysaccharidosis VI

scientific article published on 13 September 2012
Non-erythropoietic erythropoietin derivatives protect from light-induced and genetic photoreceptor degeneration.

scientific article published on 19 March 2011
Combined rod and cone transduction by adeno-associated virus 2/8.

scientific article published on 30 October 2013
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
Adeno-associated viral vectors for retinal gene transfer and treatment of retinal diseases

scientific article
Gene therapy of inherited retinal degenerations: prospects and challenges

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

scientific article published on April 2004
Cross-talk between androgen receptor/filamin A and TrkA regulates neurite outgrowth in PC12 cells

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

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

scientific article
The future looks brighter after 25 years of retinal gene therapy

scientific article published on 21 August 2017
Retinal transduction profiles by high-capacity viral vectors

scientific article published on 3 July 2014
In vivo quantitative noninvasive imaging of gene transfer by single-photon emission computerized tomography.

scientific article published in February 2003
Noninvasive repetitive imaging of somatostatin receptor 2 gene transfer with positron emission tomography.

scientific article published on 11 January 2011
Impact of age at administration, lysosomal storage, and transgene regulatory elements on AAV2/8-mediated rat liver transduction

scientific article (publication date: 2012)
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
Gene Therapy of ABCA4-Associated Diseases

scientific article published on 8 January 2015
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
Fighting Blindness with Adeno-Associated Virus Serotype 8

scientific article published on October 1, 2011
Novel Adeno-associated Viruses Derived From Pig Tissues Transduce Most Major Organs in Mice

scientific article published on October 22, 2014
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
AAV-mediated gene supply for treatment of degenerative and neovascular retinal diseases.

scientific article published on October 2010
Autonomic dysfunction in children with Hirschsprung's disease

scientific article published in 1999
Recombinant vectors based on porcine adeno-associated viral serotypes transduce the murine and pig retina

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

scientific article published on 01 May 2019
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
An in vivo doxycycline-controlled expression system for functional studies of the retina

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

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