Alessandro Fraldi

Telethon Institute of Genetics and Medicine, Italy 
Lysosomal storage disease, autophagy, lysosome, gene therapy, molecular therapy
"Alessandro Fraldi"
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Sorrentino NC, Cacace V, De Risi M, et al. (2019) Enhancing the Therapeutic Potential of Sulfamidase for the Treatment of Mucopolysaccharidosis IIIA. Molecular Therapy. Methods & Clinical Development. 15: 333-342
Fraldi A, Serafini M, Sorrentino NC, et al. (2018) Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches. Italian Journal of Pediatrics. 44: 130
Zito E, Fraldi A, Pepe S, et al. (2016) Sulfatase activities are regulated by the interaction of the sulfatase-modifying factor 1 with SUMF2. Embo Reports. 17: 1901
Sambri I, D'Alessio R, Ezhova Y, et al. (2016) Lysosomal dysfunction disrupts presynaptic maintenance and restoration of presynaptic function prevents neurodegeneration in lysosomal storage diseases. Embo Molecular Medicine
Sorrentino NC, Fraldi A. (2016) Brain Targeting in MPS-IIIA. Pediatric Endocrinology Reviews : Per. 13: 630-8
Fraldi A, Klein AD, Medina DL, et al. (2016) Brain Disorders Due to Lysomal Dysfunction. Annual Review of Neuroscience
Sorrentino NC, Maffia V, Strollo S, et al. (2015) A comprehensive map of CNS transduction by eight recombinant adeno-associated virus serotypes upon cerebrospinal fluid administration in pigs. Molecular Therapy : the Journal of the American Society of Gene Therapy
Lambertini C, Ventrella D, Barone F, et al. (2015) Transdermal spinal catheter placement in piglets: Description and validation of the technique. Journal of Neuroscience Methods. 255: 17-21
Romagnoli N, Ventrella D, Giunti M, et al. (2014) Access to cerebrospinal fluid in piglets via the cisterna magna: optimization and description of the technique. Laboratory Animals. 48: 345-8
Tardieu M, Zérah M, Husson B, et al. (2014) Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I/II trial. Human Gene Therapy. 25: 506-16
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