Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I mice-Reference-Cited by-同舟云学术

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Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I mice

Published:2006-02-16 Issue:11 Volume:13 Page:917-925
ISSN:0969-7128
Container-title:Gene Therapy
language:en
Short-container-title:Gene Ther

Author:

Watson G,Bastacky J,Belichenko P,Buddhikot M,Jungles S,Vellard M,Mobley W C,Kakkis E

Publisher

Springer Science and Business Media LLC

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.nature.com/articles/3302735.pdf

Reference44 articles.

1. Neufeld EF, Muenzer J . The Mucopolysaccharidoses. McGraw-Hill: New York, 2001 pp 3421–3452.

2. Peters C, Shapiro EG, Anderson J, Henslee-Downey PJ, Klemperer MR, Cowan MJ et al. Hurler syndrome: II. Outcome of HLA-genotypically identical sibling and HLA-haploidentical related donor bone marrow transplantation in fifty-four children. The Storage Disease Collaborative Study Group. Blood 1998; 91: 2601–2608.

3. Peters C, Balthazor M, Shapiro EG, King RJ, Kollman C, Hegland JD et al. Outcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome. Blood 1996; 87: 4894–4902.

4. Staba SL, Escolar ML, Poe M, Kim Y, Martin PL, Szabolcs P et al. Cord-blood transplants from unrelated donors in patients with Hurler's syndrome. N Engl J Med 2004; 350: 1960–1969.

5. Kakkis ED, Muenzer J, Tiller GE, Waber L, Belmont J, Passage M et al. Enzyme-replacement therapy in mucopolysaccharidosis I. N Engl J Med 2001; 344: 182–188.

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