Ex Vivo and In Vivo Gene Therapy for Mucopolysaccharidoses: State of the Art.

Cost of Illness Enzyme Replacement Therapy / methods Genetic Therapy / methods Hematopoietic Stem Cell Transplantation / methods Humans Mucopolysaccharidoses / complications
Enzyme replacement therapy Exvivo gene therapy Hematopoietic stem cell transplantation Invivo gene therapy Mucopolysaccharidoses

Journal

Hematology/oncology clinics of North America
ISSN: 1558-1977
Titre abrégé: Hematol Oncol Clin North Am
Pays: United States
ID NLM: 8709473

Informations de publication

Date de publication:
08 2022
Historique:
pubmed: 1 7 2022
medline: 20 7 2022
entrez: 30 6 2022
Statut: ppublish

Résumé

Enzyme replacement therapy (ERT) and allogeneic hematopoietic stem cell transplantation (HSCT) are standard treatments for some mucopolysaccharidoses. Nevertheless, ERT is not curative, and HSCT is associated with significant mortality and morbidity, leaving a substantial disease burden of brain and skeletal manifestations. To overcome these limitations, different gene therapy (GT) strategies are under preclinical and clinical development. Data from ex-vivo GT clinical trials have demonstrated encouraging biochemical and early clinical outcomes. In-vivo GT, based on local brain delivery or systemic intravenous injections, resulted in biochemical and clinical stabilization of the disease.

Identifiants

DOI: 10.1016/j.hoc.2022.03.012 PMID: 35773049
pubmed: 35773049
pii: S0889-8588(22)00033-8
doi: 10.1016/j.hoc.2022.03.012
pii:
doi:

Types de publication

Journal Article Review Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

865-878

Informations de copyright

Copyright © 2022 Elsevier Inc. All rights reserved.

Auteurs

Giulia Consiglieri (G)

Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan, Italy; Department of Biomedicine and Prevention, University of Rome Tor Vergata, Rome, Italy.

Maria Ester Bernardo (ME)

Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan, Italy; Vita-Salute San Raffaele University, Milan, Italy.

Nicola Brunetti-Pierri (N)

Department of Translational Medicine, Federico II University, Naples, Italy; Telethon Institute of Genetics and Medicine, Pozzuoli, Naples, Italy; Scuola Superiore Meridionale, School for Advanced Studies, Naples, Italy.

Alessandro Aiuti (A)

Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan, Italy; Vita-Salute San Raffaele University, Milan, Italy. Electronic address: aiuti.alessandro@hsr.it.

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Classifications MeSH

questionsmedicales.fr › Environnement et santé publique › Santé publique › Méthodes épidémiologiques › Collecte de données › Enquêtes et questionnaires › Enquêtes de santé › Indicateurs d'état de santé › Coûts indirects de la maladie › Ex Vivo and In Vivo Gene Therapy for...
questionsmedicales.fr › Thérapeutique › Traitement médicamenteux › Thérapie enzymatique › Thérapie enzymatique substitutive › Ex Vivo and In Vivo Gene Therapy for...
questionsmedicales.fr › Techniques d'investigation › Techniques génétiques › Génie génétique › Thérapie génétique › Ex Vivo and In Vivo Gene Therapy for...
questionsmedicales.fr › Procédures de chirurgie opératoire › Transplantation › Transplantation cellulaire › Transplantation de cellules souches › Transplantation de cellules souches hématopoïétiques › Ex Vivo and In Vivo Gene Therapy for...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Ex Vivo and In Vivo Gene Therapy for...
questionsmedicales.fr › Maladies métaboliques et nutritionnelles › Maladies métaboliques › Erreurs innées du métabolisme › Maladies lysosomiales › Mucopolysaccharidoses › Ex Vivo and In Vivo Gene Therapy for...