Nicolina C. Sorrentino

Naples, Italy

Last Updated: 09/26/2026

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Found 30 publications

TRPML1 agonists synergize with enzyme replacement therapy in fibroblasts from Pompe disease patients.

TRPML1 agonists synergize with enzyme replacement therapy in fibroblasts from Pompe disease patients.

Journal: Journal of translational medicine

Published: March 10, 2026

Retina-specific long non-coding RNAs associated with inherited retinal disease genes.

Retina-specific long non-coding RNAs associated with inherited retinal disease genes.

Journal: Cellular and molecular life sciences : CMLS

Published: February 13, 2026

A coordinated transcriptional program controls de novo Golgi biogenesis.

A coordinated transcriptional program controls de novo Golgi biogenesis.

Journal: The EMBO journal

Published: November 17, 2025

Commentary: Lysosomal enzymes engineered to cross the blood-brain barrier are reshaping the therapeutic landscape of neuronopathic mucopolysaccharidoses.

Commentary: Lysosomal enzymes engineered to cross the blood-brain barrier are reshaping the therapeutic landscape of neuronopathic mucopolysaccharidoses.

Journal: Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics

Published: October 16, 2025

Hepatocyte delivery of miR-34b/c reduces hepatic stellate cell activation and improves liver fibrosis.

Hepatocyte delivery of miR-34b/c reduces hepatic stellate cell activation and improves liver fibrosis.

Journal: Molecular therapy. Nucleic acids

Published: July 21, 2024

Anti-amyloid treatment is broadly effective in neuronopathic mucopolysaccharidoses and synergizes with gene therapy in MPS-IIIA.

Anti-amyloid treatment is broadly effective in neuronopathic mucopolysaccharidoses and synergizes with gene therapy in MPS-IIIA.

Journal: Molecular therapy : the journal of the American Society of Gene Therapy

Published: May 03, 2024

Safe and effective liver-directed AAV-mediated homology-independent targeted integration in mouse models of inherited diseases.

Safe and effective liver-directed AAV-mediated homology-independent targeted integration in mouse models of inherited diseases.

Journal: Cell reports. Medicine

Published: October 04, 2023

Role of trafficking protein particle complex 2 in medaka development.

Role of trafficking protein particle complex 2 in medaka development.

Journal: Traffic (Copenhagen, Denmark)

Published: August 07, 2023

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

Frequency-Dependent Squeezed Vacuum Source for the Advanced Virgo Gravitational-Wave Detector.

Frequency-Dependent Squeezed Vacuum Source for the Advanced Virgo Gravitational-Wave Detector.

Journal: Physical review letters

Published: February 12, 2023

The Amyloid Inhibitor CLR01 Relieves Autophagy and Ameliorates Neuropathology in a Severe Lysosomal Storage Disease.

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

TFEB and TFE3 drive kidney cystogenesis and tumorigenesis.

TFEB and TFE3 drive kidney cystogenesis and tumorigenesis.

Journal: EMBO molecular medicine

Published: September 13, 2022

Showing 1-12 of 30

Last Updated: 09/26/2026