Publications by authors named "Novella Tedesco"
Gene therapy of Usher syndrome type 1B (USH1B) due to mutations in the large () gene is limited by the packaging capacity of adeno-associated viral (AAV) vectors. To overcome this, we have previously developed dual AAV8 vectors which encode human (dual AAV8.).
View Article and Find Full Text PDFBACKGROUND: Mucopolysaccharidosis type VI (MPS VI) is an inherited multisystem lysosomal disorder due to arylsulfatase B (ARSB) deficiency that leads to widespread accumulation of glycosaminoglycans (GAG), which are excreted in increased amounts in urine. MPS VI is characterized by progressive dysostosis multiplex, connective tissue and cardiac involvement, and hepatosplenomegaly. Enzyme replacement therapy (ERT) is available but requires life-long and costly intravenous infusions; moreover, it has limited efficacy on diseased skeleton and cardiac valves, compromised pulmonary function, and corneal opacities.
View Article and Find Full Text PDFArticle Synopsis
- - One major goal of gene transfer therapy is to achieve long-lasting expression of therapeutic genes in specialized cells, with adeno-associated virus (AAV) vectors being recognized as effective for this purpose thanks to recent approvals like Luxturna and Zolgensma.
- - Despite their success, AAV vectors face limitations due to widespread pre-existing anti-AAV antibodies in the human population, which can neutralize these therapeutic vectors after initial exposure.
- - Strategies to address these immunogenicity issues are being explored, with preclinical and clinical data providing insights necessary for improving safety and effectiveness of AAV-based gene therapies for broader patient populations.
Mucopolysaccharidosis type IIIA (MPS-IIIA) is a lysosomal storage disorder (LSD) caused by inherited defect of sulfamidase, a lysosomal sulfatase. MPS-IIIA is one of the most common and severe forms of LSDs with CNS involvement. Presently there is no cure.
View Article and Find Full Text PDF