Evaluation of AAV vectors with tissue-specific or ubiquitous promoters in a mouse model of mucopolysaccharidosis type IVA-Reference-Cited by-同舟云学术

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Evaluation of AAV vectors with tissue-specific or ubiquitous promoters in a mouse model of mucopolysaccharidosis type IVA

Published:2025-06 Issue:2 Volume:33 Page:101447
ISSN:2329-0501
Container-title:Molecular Therapy Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy Methods & Clinical Development

Author:

Khan Shaukat A.,Álvarez Jose Victor,Nidhi F.N.U.,Benincore-Florez Eliana,Tomatsu Shunji

Publisher

Elsevier BV

Reference68 articles.

1. Morquio's syndrome: deficiency of a chondroitin sulfate N-acetylhexosamine sulfate sulfatase;Matalon;Biochem. Biophys. Res. Commun.,1974

2. The enzymic defects in Morquio and Maroteaux-Lamy syndrome;Dorfman;Adv. Exp. Med. Biol.,1976

3. N-acetylgalactosamine-6-sulfate sulfatase in man. Absence of the enzyme in Morquio disease;Singh;J. Clin. Investig.,1976

4. Deficiencies of glucosamine-6-sulfate or galactosamine-6-sulfate sulfatases are responsible for different mucopolysaccharidoses;Di Ferrante;Science,1978

5. International Morquio A Registry: clinical manifestation and natural course of Morquio A disease;Montano;J. Inherit. Metab. Dis.,2007

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