An empowered, clinically viable hematopoietic stem cell gene therapy for the treatment of multisystemic mucopolysaccharidosis type II-Reference-Cited by-同舟云学术

An empowered, clinically viable hematopoietic stem cell gene therapy for the treatment of multisystemic mucopolysaccharidosis type II

Published:2024-03 Issue:3 Volume:32 Page:619-636
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Das Sabyasachi,Rruga Fatlum,Montepeloso Annita^ORCID,Dimartino Agnese,Spadini Silvia,Corre Guillaume,Patel Janki,Cavalca Eleonora,Ferro Francesca,Gatti Alessandra,Milazzo Rita,Galy Anne,Politi Letterio S.,Rizzardi Gian Paolo,Vallanti Giuliana,Poletti Valentina,Biffi Alessandra^ORCID

Funder

Medizinischen Hochschule Hannover

Publisher

Elsevier BV

Reference54 articles.

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2. Natural progression of neurological disease in mucopolysaccharidosis type II;Holt;Pediatrics,2011

3. A phase I/II study of intrathecal idursulfase-IT in children with severe mucopolysaccharidosis II;Muenzer;Genet. Med.,2016

4. Hematopoietic cell transplantation for mucopolysaccharidosis patients is safe and effective: results after implementation of international guidelines;Aldenhoven;Biol. Blood Marrow Transpl.,2015

5. Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning;Boelens;Blood,2013

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