TERAPIA GENICA DEI DIFETTI CONGENITI DEL METABOLISMO EPATICO
- 3 Anni 2009/2012
- 290.000€ Totale Fondi
Descrizione non disponibile
Pubblicazioni Scientifiche
- 2013-03-01 EMBO MOLECULAR MEDICINE
Gene transfer of master autophagy regulator TFEB results in clearance of toxic protein and correction of hepatic disease in alpha-1-anti-trypsin deficiency
- 2009-05-01 HUMAN GENE THERAPY
Bioengineered Factor IX Molecules with Increased Catalytic Activity Improve the Therapeutic Index of Gene Therapy Vectors for Hemophilia B
- 2012-10-01 HUMAN GENE THERAPY
Sustained Reduction of Hyperbilirubinemia in Gunn Rats After Adeno-Associated Virus-Mediated Gene Transfer of Bilirubin UDP-Glucuronosyltransferase Isozyme 1A1 to Skeletal Muscle
- 2012-12-01 JOURNAL OF PEDIATRIC GASTROENTEROLOGY AND NUTRITION
Autosomal Dominant Menetrier-like Disease
