Vincenzo Cacace
Naples, Italy
Last Updated: 09/26/2026
Save publications for later
Sign Up
Not sure about your diagnosis?
Check Your Symptoms
Found 8 publications
Increased expression or activation of TRPML1 reduces hepatic storage of toxic Z alpha-1 antitrypsin.
Increased expression or activation of TRPML1 reduces hepatic storage of toxic Z alpha-1 antitrypsin.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: April 19, 2023
The Amyloid Inhibitor CLR01 Relieves Autophagy and Ameliorates Neuropathology in a Severe Lysosomal Storage Disease.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: October 19, 2022
New mouse models with hypomorphic SUMF1 variants mimic attenuated forms of multiple sulfatase deficiency.
Journal: Journal of inherited metabolic disease
Published: February 21, 2022
Fluoxetine ameliorates mucopolysaccharidosis type IIIA.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: June 22, 2021
The Amyloid Inhibitor CLR01 Relieves Autophagy and Ameliorates Neuropathology in a Severe Lysosomal Storage Disease.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: October 18, 2019
Enhancing the Therapeutic Potential of Sulfamidase for the Treatment of Mucopolysaccharidosis IIIA.
Journal: Molecular therapy. Methods & clinical development
Published: June 06, 2019
Lysosomal dysfunction disrupts presynaptic maintenance and restoration of presynaptic function prevents neurodegeneration in lysosomal storage diseases.
Journal: EMBO molecular medicine
Published: November 25, 2016
A Comprehensive Map of CNS Transduction by Eight Recombinant Adeno-associated Virus Serotypes Upon Cerebrospinal Fluid Administration in Pigs.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: June 01, 2015
Showing 1-8 of 8
Last Updated: 09/26/2026