GENE THERAPY STRATEGIES FOR INHERITED SEVERE PHOTORECEPTOR DISEASES
- 5 Years 2006/2011
- 865.000€ Total Award
Inherited retinal diseases are frequent causes of blindness for which no cure is currently available.
Ideally, transfer of the genes lacking in inherited retinal diseases might restore or preserve visual function. The goal of our project is to design and test gene-based therapeutic strategies to correct severe inherited retinal diseases which affect a specific cell type in the retina, the photoreceptor.
Since the results of this study may provide novel therapeutic strategies for inherited retinal diseases we propose to collect a series of Italian patients to define their molecular diagnosis and clinical characteristics.
Scientific Publications
- 2004 MOLECULAR THERAPY
Systemic but not intraocular Epo gene transfer protects the retina from light- and genetic-induced degeneration
- 2008 NEW ENGLAND JOURNAL OF MEDICINE
Safety and efficacy of gene transfer for Leber's congenital amaurosis
- 2009 LANCET
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
- 2004 MOLECULAR VISION
Effect of over-expression of pigment epithelium derived factor (PEDF) on developing retinal vasculature in the mouse
- 2010 MOLECULAR THERAPY
Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration
- 2010 PLOS ONE
Mouse Embryonic Retina Delivers Information Controlling Cortical Neurogenesis
- 2009 MOLECULAR THERAPY
AAV-mediated Tyrosinase Gene Transfer Restores Melanogenesis and Retinal Function in a Model of Oculo-cutaneous Albinism Type I (OCA1)
- 2006 MOLECULAR THERAPY
Inhibition of ocular neovascularization by hedgehog blockade
- 2009 HUMAN MOLECULAR GENETICS
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
- 2007 JOURNAL OF VIROLOGY
Novel adeno-associated virus serotypes efficiently transduce murine Photoreceptors
- 2009 TRENDS IN MOLECULAR MEDICINE
Ocular gene therapy: current progress and future prospects
