Encapsulated engineered myoblasts can cure Hurler syndrome: preclinical experiments in the mouse model-Reference-Cited by-同舟云学术

Encapsulated engineered myoblasts can cure Hurler syndrome: preclinical experiments in the mouse model

Published:2011-06-30 Issue:4 Volume:19 Page:355-364
ISSN:0969-7128
Container-title:Gene Therapy
language:en
Short-container-title:Gene Ther

Author:

Piller Puicher E,Tomanin R,Salvalaio M,Friso A,Hortelano G,Marin O,Scarpa M

Publisher

Springer Science and Business Media LLC

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.nature.com/articles/gt201194.pdf

Reference31 articles.

1. Neufeld EF, Muenzer J . The mucopolysaccharidoses. In: The Metabolic and Molecular Bases of Inherited Disease, Vol. 3. McGraw-Hill Professional Publishing: New York, 2001, pp 3421–3452.

2. Illsinger S, Lucke T, Hartmann H, Mengel E, Muller-Forell W, Donnerstag F et al. Scheie syndrome: enzyme replacement therapy does not prevent progression of cervical myelopathy due to spinal cord compression. J Inherit Metab Dis. Short Report #190 (2009), Published online 4 November 2009; doi:10.1007/s10545-009-1265-y.

3. Hobbs JR, Hugh-Jones K, Barrett AJ, Byrom N, Chambers D, Henry K et al. Reversal of clinical features of Hurler's disease and biochemical improvement after treatment by bone-marrow transplantation. Lancet 1981; 2: 709–712.

4. Peters C, Steward CG . Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines. Bone Marrow Transplant 2003; 31: 229–239.

5. Wraith JE . Enzyme replacement therapy in mucopolysaccharidosis type I: progress and emerging difficulties. J Inherit Metab Dis 2001; 24: 245–250.

Cited by 15 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Bioactive Glial-Derived Neurotrophic Factor from a Safe Injectable Collagen–Alginate Composite Gel Rescues Retinal Photoreceptors from Retinal Degeneration in Rabbits;Marine Drugs;2024-08-30

2. Transplantable cell-encapsulation device using a semipermeable ethylene-vinyl alcohol copolymer membrane in a mouse diabetic model;Journal of Bioscience and Bioengineering;2023-11

3. Encapsulation: Advances in cell encapsulation;Principles of Biomaterials Encapsulation : Volume One;2023

4. Drosophila D-idua Reduction Mimics Mucopolysaccharidosis Type I Disease-Related Phenotypes;Cells;2021-12-31

5. Pre-clinical Mouse Models of Neurodegenerative Lysosomal Storage Diseases;Frontiers in Molecular Biosciences;2020-04-15