Replacing the enzyme alpha-L-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I.
Dierenfeld AD, McEntee MF, Vogler CA, Vite CH, Chen AH, Passage M, Le S, Shah S, Jens JK, Snella EM, Kline KL, Parkes JD, Ware WA, Moran LE, Fales-Williams AJ, Wengert JA, Whitley RD, Betts DM, Boal AM, Riedesel EA, Gross W, Ellinwood NM, Dickson PI.
Dierenfeld AD, et al. Among authors: chen ah.
Sci Transl Med. 2010 Dec 1;2(60):60ra89. doi: 10.1126/scitranslmed.3001380.
Sci Transl Med. 2010.
PMID: 21123810
Free PMC article.