Nicolina Cristina Sorrentino

Telethon Institute Of Genetics And Medicine — Italy

Medicine · Health Sciences

15h-index854citations32works3.02yr avg
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Research Topics

Lysosomal Storage Disorders Research(14), Virus-based gene therapy research(7), Cellular transport and secretion(7), CRISPR and Genetic Engineering(3), Trypanosoma species research and implications(3)

Publications32 total

Suppression of ADPKD Alters Epithelial Cell Transcription Program
Journal of the American Society of Nephrology·2025
Commentary: Lysosomal enzymes engineered to cross the blood-brain barrier are reshaping the therapeutic landscape of neuronopathic mucopolysaccharidoses
Neurotherapeutics·2025Open Access
<i>De novo</i> Golgi biogenesis requires coordinated transactivation of a Golgi regulon
2025Open Access
Hepatocyte delivery of miR-34b/c reduces hepatic stellate cell activation and improves liver fibrosis
Molecular Therapy — Nucleic Acids·2025Open Access
Safe and effective liver-directed AAV-mediated homology-independent targeted integration in mouse models of inherited diseases
Cell Reports Medicine·2024· 15 citedOpen Access
Role of trafficking protein particle complex 2 in medaka development
Traffic·2023· 2 citedOpen Access
Author response for "Role of trafficking protein particle complex 2 in medaka development"
2023
Role of trafficking protein particle complex 2 in medaka development
2023· 2 citedOpen Access
Increased expression or activation of TRPML1 reduces hepatic storage of toxic Z alpha-1 antitrypsin
Molecular Therapy·2023· 5 citedOpen Access
<scp>TFEB</scp> and <scp>TFE3</scp> drive kidney cystogenesis and tumorigenesis
EMBO Molecular Medicine·2023· 54 citedOpen Access
New mouse models with hypomorphic <i>SUMF1</i> variants mimic attenuated forms of multiple sulfatase deficiency
Journal of Inherited Metabolic Disease·2022· 8 citedOpen Access
Lysosomal fusion and <scp>SNARE</scp> function are impaired by cholesterol accumulation in lysosomal storage disorders
The EMBO Journal·2022· 5 citedOpen Access
The Amyloid Inhibitor CLR01 Relieves Autophagy and Ameliorates Neuropathology in a Severe Lysosomal Storage Disease
Molecular Therapy·2022· 1 citedOpen Access
Fluoxetine ameliorates mucopolysaccharidosis type IIIA
Molecular Therapy·2022· 27 citedOpen Access
Retinal Degeneration in MPS-IIIA Mouse Model
Frontiers in Cell and Developmental Biology·2020· 19 citedOpen Access
The Amyloid Inhibitor CLR01 Relieves Autophagy and Ameliorates Neuropathology in a Severe Lysosomal Storage Disease
Molecular Therapy·2020· 38 citedOpen Access
Enhancing the Therapeutic Potential of Sulfamidase for the Treatment of Mucopolysaccharidosis IIIA
Molecular Therapy — Methods & Clinical Development·2019· 16 citedOpen Access
Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches
˜The œItalian Journal of Pediatrics/Italian journal of pediatrics·2018· 47 citedOpen Access
Effects of non‐euphoric plant cannabinoids on muscle quality and performance of dystrophic mdx mice
British Journal of Pharmacology·2018· 72 citedOpen Access
Enhanced Version of Human Sulfamidase Significantly Ameliorates CNS Pathology When Delivered to the MPS-IIIA Mice by AAV-Mediated Intra-CSF Injection
Archivio istituzionale della ricerca (Alma Mater Studiorum Università di Bologna)·2018
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Frequent Co-authors

Alessandro Fraldi(19), Edoardo Nusco(12), Vincenzo Cacace(10), Elvira De Leonibus(8), Veronica Maffia(8), Elena Polishchuk(7), Domenico Ventrella(7), Andrea Ballabio(6), Rossella De Cegli(6), Maria Laura Bacci(6), Maria De Risi(5), Alberto Auricchio(5), Enrico Maria Surace(5), Sandra Strollo(5), Carmine Spampanato(4), Irene Sambri(4), Teresa Giuliano(4), Noemi Romagnoli(4), Massimo Giunti(4), Francesco Dondi(4)