Mostrar el registro sencillo del ítem

dc.contributor.authorCastro, María José dept_BR
dc.contributor.authorToro, Mireia delpt_BR
dc.contributor.authorGiugliani, Robertopt_BR
dc.contributor.authorCouce, María Luzpt_BR
dc.date.accessioned2022-01-27T04:34:01Zpt_BR
dc.date.issued2021pt_BR
dc.identifier.issn1422-0067pt_BR
dc.identifier.urihttp://hdl.handle.net/10183/234538pt_BR
dc.description.abstractThe need for long-lasting and transformative therapies for mucopolysaccharidoses (MPS) cannot be understated. Currently, many forms of MPS lack a specific treatment and in other cases available therapies, such as enzyme replacement therapy (ERT), do not reach important areas such as the central nervous system (CNS). The advent of newborn screening procedures represents a major step forward in early identification and treatment of individuals with MPS. However, the treatment of brain disease in neuronopathic MPS has been a major challenge to date, mainly because the blood brain barrier (BBB) prevents penetration of the brain by large molecules, including enzymes. Over the last years several novel experimental therapies for neuronopathic MPS have been investigated. Gene therapy and gene editing constitute potentially curative treatments. However, despite recent progress in the field, several considerations should be taken into account. This review focuses on the state of the art of in vivo and ex vivo gene therapy-based approaches targeting the CNS in neuronopathic MPS, discusses clinical trials conducted to date, and provides a vision for the future implications of these therapies for the medical community. Recent advances in the field, as well as limitations relating to efficacy, potential toxicity, and immunogenicity, are also discussed.en
dc.format.mimetypeapplication/pdfpt_BR
dc.language.isoengpt_BR
dc.relation.ispartofInternational journal of molecular sciences. Basel. Vol. 22 (2021), 9200, 17 p.pt_BR
dc.rightsOpen Accessen
dc.subjectMucopolysaccharidosesen
dc.subjectMucopolissacaridosespt_BR
dc.subjectGene therapyen
dc.subjectSistema nervoso centralpt_BR
dc.subjectViral vectorsen
dc.subjectTerapia genéticapt_BR
dc.subjectAdeno-associated virusen
dc.subjectBarreira hematoencefálicapt_BR
dc.subjectLentivirusen
dc.subjectRevisãopt_BR
dc.subjectCentral nervous systemen
dc.subjectBlood brain barrieren
dc.titleGene therapy for neuronopathic mucopolysaccharidoses : state of the artpt_BR
dc.typeArtigo de periódicopt_BR
dc.identifier.nrb001136219pt_BR
dc.type.originEstrangeiropt_BR


Ficheros en el ítem

Thumbnail
   

Este ítem está licenciado en la Creative Commons License

Mostrar el registro sencillo del ítem