TERAPIA GENICA DEI DIFETTI CONGENITI DEL METABOLISMO EPATICO

Pubblicazioni Scientifiche

• 2013 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

• 2012 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

• 2009 HUMAN GENE THERAPY

  Bioengineered Factor IX Molecules with Increased Catalytic Activity Improve the Therapeutic Index of Gene Therapy Vectors for Hemophilia B

• 2012 JOURNAL OF PEDIATRIC GASTROENTEROLOGY AND NUTRITION

  Autosomal Dominant Menetrier-like Disease