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List of works by Enrico M. Surace

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

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

scientific article

Access to cerebrospinal fluid in piglets via the cisterna magna: optimization and description of the technique

scientific article published on 26 June 2014

Adeno-associated viral vectors for retinal gene transfer.

scientific article published on November 2003

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

scientific article

Allele-specific editing ameliorates dominant retinitis pigmentosa in a transgenic mouse model

scientific article published on 27 January 2021

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

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

scientific article published on 30 October 2013

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

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

scientific article

Gene therapy of inherited retinal degenerations: prospects and challenges

scientific article

In utero gene therapy rescues vision in a murine model of congenital blindness

scientific article published in February 2004

Inhibition of ocular neovascularization by hedgehog blockade.

scientific article

MicroRNA-restricted transgene expression in the retina

scientific article

Molecular and clinical characterization of albinism in a large cohort of Italian patients

scientific article

Mouse embryonic retina delivers information controlling cortical neurogenesis

scientific article

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

scientific article published on 19 March 2011

Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors

scientific article

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

scientific article

Rhodopsin targeted transcriptional silencing by DNA-binding

scientific article published on 14 March 2016

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

scientific article

Sulfatase modifying factor 1 trafficking through the cells: from endoplasmic reticulum to the endoplasmic reticulum

scientific article

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

scientific article published in November 2004

The ocular albinism type 1 (OA1) G-protein-coupled receptor functions with MART-1 at early stages of melanogenesis to control melanosome identity and composition

scientific article

The ocular albinism type 1 (OA1) gene controls melanosome maturation and size

scientific article published in December 2005

The ocular albinism type 1 protein, an intracellular G protein-coupled receptor, regulates melanosome transport in pigment cells

scientific article

The role of optical coherence tomography in an atypical case of oculocutaneous albinism: a case report.

scientific article published on January 2012

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

Transdermal spinal catheter placement in piglets: Description and validation of the technique

scientific article

Versatility of AAV vectors for retinal gene transfer

scientific article published on 17 October 2007

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

scientific article

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