Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type I-Reference-Cited by-同舟云学术

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Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type I

Published:2014-09-29 Issue:41 Volume:111 Page:14894-14899
ISSN:0027-8424
Container-title:Proceedings of the National Academy of Sciences
language:en
Short-container-title:Proceedings of the National Academy of Sciences

Author:

Hinderer C.,Bell P.,Gurda B. L.,Wang Q.,Louboutin J.-P.,Zhu Y.,Bagel J.,O'Donnell P.,Sikora T.,Ruane T.,Wang P.,Haskins M. E.,Wilson J. M.

Publisher

Proceedings of the National Academy of Sciences

Subject

Multidisciplinary

Reference33 articles.

1. Radiological and clinical characterization of the lysosomal storage disorders: non-lipid disorders

2. Usefulness of bone marrow transplantation in the Hurler syndrome

3. The prevalence of and survival in Mucopolysaccharidosis I: Hurler, Hurler-Scheie and Scheie syndromes in the UK

4. Overexpression of the Human Lysosomal Enzyme α-L-Iduronidase in Chinese Hamster Ovary Cells

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1. Lentiviral Gene Therapy for Mucopolysaccharidosis II with Tagged Iduronate 2-Sulfatase Prevents Life-Threatening Pathology in Peripheral Tissues But Fails to Correct Cartilage;Human Gene Therapy;2024-02-02

2. In vivo adenine base editing corrects newborn murine model of Hurler syndrome;Molecular Biomedicine;2023-02-23

3. Neurologic Recovery in MPS I and MPS II Mice by AAV9-Mediated Gene Transfer to the CNS After the Development of Cognitive Dysfunction;Human Gene Therapy;2023-01-01

4. First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B;Molecular Therapy;2022-12

5. Liver-directed gene therapy corrects neurologic disease in a murine model of mucopolysaccharidosis type I-Hurler;Molecular Therapy - Methods & Clinical Development;2022-06

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