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List of works by Pasquale Piccolo

Autosomal Dominant Ménétrier-like Disease

scientific article published in December 2012

Balloon Catheter Delivery of Helper-dependent Adenoviral Vector Results in Sustained, Therapeutic hFIX Expression in Rhesus Macaques ⬇️

scientific article published on July 24, 2012

CHOP and c-JUN upregulate the mutant Z alpha-1 antitrypsin, exacerbating its aggregation and liver proteotoxicity

scientific article published on 28 July 2020

Cutis laxa and fatal pulmonary hypertension: a newly recognized syndrome?

scientific article

Gene therapy for inherited diseases of liver metabolism.

scientific article published on April 2015

Gene transfer of master autophagy regulator TFEB results in clearance of toxic protein and correction of hepatic disease in alpha-1-anti-trypsin deficiency

scientific article published on 4 February 2013

Helper-dependent adenoviral vectors for liver-directed gene therapy of primary hyperoxaluria type 1.

scientific article

Improved efficacy and reduced toxicity by ultrasound-guided intrahepatic injections of helper-dependent adenoviral vector in Gunn rats

scientific article

Intrathecal Injection of Helper-Dependent Adenoviral Vectors Results in Long-Term Transgene Expression in Neuroependymal Cells and Neurons ⬇️

scientific article published on March 18, 2011

Liver-directed gene-based therapies for inborn errors of metabolism

scientific article published on 03 September 2020

Retinal transduction profiles by high-capacity viral vectors

scientific article published on 3 July 2014

SMAD4 mutations causing Myhre syndrome result in disorganization of extracellular matrix improved by losartan

scientific article published on 08 January 2014

SR-A and SREC-I are Kupffer and endothelial cell receptors for helper-dependent adenoviral vectors

scientific article published on 29 January 2013

SR-A and SREC-I binding peptides increase HDAd-mediated liver transduction.

scientific article

Slc7a7 disruption causes fetal growth retardation by downregulating Igf1 in the mouse model of lysinuric protein intolerance

scientific journal article

Supravalvular aortic stenosis: elastin arteriopathy

scientific article

Terminal osseous dysplasia with pigmentary defects (TODPD): Follow-up of the first reported family, characterization of the radiological phenotype, and refinement of the linkage region

scientific article published in July 2010

The growth hormone-insulin-like growth factor axis in glycogen storage disease type 1: evidence of different growth patterns and insulin-like growth factor levels in patients with glycogen storage disease type 1a and 1b.

scientific article published on 21 December 2009

Wilson disease protein ATP7B utilizes lysosomal exocytosis to maintain copper homeostasis.

scientific article published on 5 June 2014

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