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Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches

Fraldi, Alessandro ; Serafini, Marta ; Sorrentino, Nicolina Cristina ; Gentner, Bernhard ; Aiuti, Alessandro ; Bernardo, Maria Ester

Italian journal of pediatrics, 2018-11, Vol.44 (Suppl 2), p.130-130, Article 130 [Periódico revisado por pares]

England: BioMed Central

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  • Título:
    Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches
  • Autor: Fraldi, Alessandro ; Serafini, Marta ; Sorrentino, Nicolina Cristina ; Gentner, Bernhard ; Aiuti, Alessandro ; Bernardo, Maria Ester
  • Assuntos: Genetic Therapy - methods ; Humans ; Mucopolysaccharidoses - therapy ; Review
  • É parte de: Italian journal of pediatrics, 2018-11, Vol.44 (Suppl 2), p.130-130, Article 130
  • Notas: ObjectType-Article-2
    SourceType-Scholarly Journals-1
    ObjectType-Feature-3
    content type line 23
    ObjectType-Review-1
  • Descrição: Mucopolysaccharidoses (MPS) are a group of lysosomal storage disorders caused by a deficiency in lysosomal enzymes catalyzing the stepwise degradation of glycosaminoglycans (GAGs). The current therapeutic strategies of enzyme replacement therapy and allogeneic hematopoietic stem cell transplantation have been reported to reduce patient morbidity and to improve their quality of life, but they are associated with persistence of residual disease burden, in particular at the neurocognitive and musculoskeletal levels. This indicates the need for more efficacious treatments capable of effective and rapid enzyme delivery to the affected organs, especially the brain and the skeleton. Gene therapy (GT) strategies aimed at correcting the genetic defect in patient cells could represent a significant improvement for the treatment of MPS when compared with conventional approaches. While in-vivo GT strategies foresee the administration of viral vector particles directly to patients with the aim of providing normal complementary DNA to the affected cells, ex-vivo GT approaches are based on the ex-vivo transduction of patient cells that are subsequently infused back. This review provides insights into the state-of-art accomplishments made with in vivo and ex vivo GT-based approaches in MPS and provide a vision for the future in the medical community.
  • Editor: England: BioMed Central
  • Idioma: Inglês
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  • Realização: ABCD-USP USP   Logos de Redes Sociais: Freepik

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