Lentiviral Gene Therapy for Mucopolysaccharidosis II with Tagged Iduronate 2-Sulfatase Prevents Life-Threatening Pathology in Peripheral Tissues But Fails to Correct Cartilage-Reference-Cited by-同舟云学术

Lentiviral Gene Therapy for Mucopolysaccharidosis II with Tagged Iduronate 2-Sulfatase Prevents Life-Threatening Pathology in Peripheral Tissues But Fails to Correct Cartilage

Published:2024-02-02 Issue: Volume: Page:
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Catalano Fabio¹²³,Vlaar Eva C.¹²³,Dammou Zina¹²³,Katsavelis Drosos¹²³,Huizer Tessa F.¹²³,Zundo Giacomo¹²³,Hoogeveen-Westerveld Marianne¹³,Oussoren Esmeralda²³,van den Hout Hannerieke J.M.P.²³,Schaaf Gerben¹²³,Pike-Overzet Karin⁴,Staal Frank J.T.⁴⁵,van der Ploeg Ans T.²³,Pijnappel W.W.M. Pim¹²³^ORCID

Affiliation:

1. Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.

2. Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.

3. Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, The Netherlands.

4. Department of Immunology, Leiden University Medical Center, Leiden, The Netherlands.

5. Department of Pediatrics, Leiden University Medical Center, Leiden, The Netherlands.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2023.177

Reference49 articles.

1. Mucopolysaccharidosis Type II: One Hundred Years of Research, Diagnosis, and Treatment

2. Aortic Root Dilatation in Mucopolysaccharidosis I–VII

3. The Carotid Intima-Media Thickness and Arterial Stiffness of Pediatric Mucopolysaccharidosis Patients Are Increased Compared to Both Pediatric and Adult Controls

4. Aortic root dilatation in patients with mucopolysaccharidoses and the impact of enzyme replacement therapy

5. Retrospective analysis of the clinical manifestations and survival of Korean patients with mucopolysaccharidosis type II: Emphasis on the cardiovascular complication and mortality cases