VERSO STUDI CLINICI DI TERAPIA GENICA DELL’OCCHIO E DEL FEGATO TRAMITE AAV
- 5 Anni 2011/2016
- 300.000€ Totale Fondi
La terapia genica per le malattie ereditarie umane inizia a raccogliere i primi successi a livello clinico. Al TIGEM ci siamo concentrati sulla terapia genica delle malattie retiniche ereditarie e degli errori congeniti del metabolismo, in quanto per queste malattie manca una terapia e anche perchè sia le malattie che gli organi interessati hanno caratteristiche favorevoli per la terapia genica. L'obiettivo generale di questo programma di ricerca è quello di giungere rapidamente a testare in clinica approcci di terapia genica mediante vettori virali adeno-associati (AAV) in pazienti affetti da una grave forma di cecità infantile e da due difetti congeniti del metabolismo in cui il fegato è l'organo bersaglio. Le malattie sono state selezionate sulla base di: I. modello recessivo di ereditarietà II. severità e favorevole rapporto rischio-beneficio III. dati preclinici che sostengono l'efficacia dei vettori AAV2/8 IV. disponibilità di pazienti. Produrremo il set completo di dati preclinici che supportano l'efficacia delle strategie di terapia genica mediata da AAV per passare poi a testarle in pazienti.
Pubblicazioni Scientifiche
- 2011-07-01 GENE THERAPY
AAV-mediated photoreceptor transduction of the pig cone-enriched retina
- 2012-02-01 GENE THERAPY
Novel adeno-associated viral vectors for retinal gene therapy
- 2014-04-01 GENE THERAPY
Efficient gene delivery to the cone-enriched pig retina by dual AAV vectors
- 2011-10-01 HUMAN GENE THERAPY
Fighting Blindness with Adeno-Associated Virus Serotype 8
- 2013-02-01 HUMAN GENE THERAPY
Gene Therapy for Mucopolysaccharidosis Type VI Is Effective in Cats Without Pre-Existing Immunity to AAV8
- 2014-07-01 HUMAN GENE THERAPY
Similar Therapeutic Efficacy Between a Single Administration of Gene Therapy and Multiple Administrations of Recombinant Enzyme in a Mouse Model of Lysosomal Storage Disease
- 2015-03-01 HUMAN GENE THERAPY
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
- 2015-04-01 HUMAN GENE THERAPY
Gene Therapy for Inherited Diseases of Liver Metabolism
- 2013-10-01 HUMAN GENE THERAPY METHODS
Improved Efficacy and Reduced Toxicity by Ultrasound-Guided Intrahepatic Injections of Helper-Dependent Adenoviral Vector in Gunn Rats
- 2014-06-01 HUMAN GENE THERAPY METHODS
Correction of Hyperbilirubinemia in Gunn Rats by Surgical Delivery of Low Doses of Helper-Dependent Adenoviral Vectors
- 2011-03-01 INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE
Molecular and Clinical Characterization of Albinism in a Large Cohort of Italian Patients
- 2011-07-01 INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE
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 βPDE Deficiency
- 2011-07-01 INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE
Evaluation of Italian Patients with Leber Congenital Amaurosis due to AIPL1 Mutations Highlights the Potential Applicability of Gene Therapy
- 2011-06-01 JOURNAL OF CLINICAL INVESTIGATION
The human visual cortex responds to gene therapy-mediated recovery of retinal function
- 2012-10-01 MOLECULAR THERAPY
Balloon Catheter Delivery of Helper-dependent Adenoviral Vector Results in Sustained, Therapeutic hFIX Expression in Rhesus Macaques
- 2017-09-01 MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT
Non-clinical Safety and Efficacy of an AAV2/8 Vector Administered Intravenously for Treatment of Mucopolysaccharidosis Type VI
- 2012-03-13 PLOS ONE
Impact of Age at Administration, Lysosomal Storage, and Transgene Regulatory Elements on AAV2/8-Mediated Rat Liver Transduction
- 2012-02-08 SCIENCE TRANSLATIONAL MEDICINE
AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness
- 2014-01-10 SCIENTIFIC REPORTS
Sensory-motor behavioral characterization of an animal model of Maroteaux-Lamy syndrome (or Mucopolysaccharidosis VI)
- 2014-10-22 SCIENTIFIC REPORTS
Novel Adeno-associated Viruses Derived From Pig Tissues Transduce Most Major Organs in Mice
