ODonnell P - Search Results

1

Krabbe disease successfully treated via monotherapy of intrathecal gene therapy.

Bradbury AM, Bagel JH, Nguyen D, Lykken EA, Pesayco Salvador J, Jiang X, Swain GP, Assenmacher CA, Hendricks IJ, Miyadera K, Hess RS, Ostrager A, ODonnell P, Sands MS, Ory DS, Shelton GD, Bongarzone ER, Gray SJ, Vite CH. Bradbury AM, et al. Among authors: odonnell p. J Clin Invest. 2020 Sep 1;130(9):4906-4920. doi: 10.1172/JCI133953. J Clin Invest. 2020. PMID: 32773406 Free PMC article.

2

Expression in blood cells may contribute to biochemical and pathological improvements after neonatal intravenous gene therapy for mucopolysaccharidosis VII in dogs.

Wang B, O'Malley TM, Xu L, Vite C, Wang P, O'Donnell PA, Ellinwood NM, Haskins ME, Ponder KP. Wang B, et al. Mol Genet Metab. 2006 Jan;87(1):8-21. doi: 10.1016/j.ymgme.2005.08.014. Epub 2005 Nov 7. Mol Genet Metab. 2006. PMID: 16275036

3

Correction of clinical manifestations of canine mucopolysaccharidosis I with neonatal retroviral vector gene therapy.

Traas AM, Wang P, Ma X, Tittiger M, Schaller L, O'donnell P, Sleeper MM, Vite C, Herati R, Aguirre GD, Haskins M, Ponder KP. Traas AM, et al. Mol Ther. 2007 Aug;15(8):1423-31. doi: 10.1038/sj.mt.6300201. Epub 2007 May 22. Mol Ther. 2007. PMID: 17519893 Free article.

4

Clinical, electrophysiological, and serum biochemical measures of progressive neurological and hepatic dysfunction in feline Niemann-Pick type C disease.

Vite CH, Ding W, Bryan C, O'Donnell P, Cullen K, Aleman D, Haskins ME, Van Winkle T. Vite CH, et al. Pediatr Res. 2008 Nov;64(5):544-9. doi: 10.1203/PDR.0b013e318184d2ce. Pediatr Res. 2008. PMID: 18614965 Free PMC article.

5

2-hydroxypropyl-beta-cyclodextrin raises hearing threshold in normal cats and in cats with Niemann-Pick type C disease.

Ward S, O'Donnell P, Fernandez S, Vite CH. Ward S, et al. Pediatr Res. 2010 Jul;68(1):52-6. doi: 10.1203/PDR.0b013e3181df4623. Pediatr Res. 2010. PMID: 20357695 Free PMC article.

6

Miglustat improves purkinje cell survival and alters microglial phenotype in feline Niemann-Pick disease type C.

Stein VM, Crooks A, Ding W, Prociuk M, O'Donnell P, Bryan C, Sikora T, Dingemanse J, Vanier MT, Walkley SU, Vite CH. Stein VM, et al. J Neuropathol Exp Neurol. 2012 May;71(5):434-48. doi: 10.1097/NEN.0b013e31825414a6. J Neuropathol Exp Neurol. 2012. PMID: 22487861 Free PMC article.

7

Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis I.

Hinderer C, Bell P, Gurda BL, Wang Q, Louboutin JP, Zhu Y, Bagel J, O'Donnell P, Sikora T, Ruane T, Wang P, Haskins ME, Wilson JM. Hinderer C, et al. Mol Ther. 2014 Dec;22(12):2018-2027. doi: 10.1038/mt.2014.135. Epub 2014 Jul 16. Mol Ther. 2014. PMID: 25027660 Free PMC article.

8

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

Hinderer C, Bell P, Gurda BL, Wang Q, Louboutin JP, Zhu Y, Bagel J, O'Donnell P, Sikora T, Ruane T, Wang P, Haskins ME, Wilson JM. Hinderer C, et al. Proc Natl Acad Sci U S A. 2014 Oct 14;111(41):14894-9. doi: 10.1073/pnas.1413645111. Epub 2014 Sep 29. Proc Natl Acad Sci U S A. 2014. PMID: 25267637 Free PMC article.

9

Intracisternal cyclodextrin prevents cerebellar dysfunction and Purkinje cell death in feline Niemann-Pick type C1 disease.

Vite CH, Bagel JH, Swain GP, Prociuk M, Sikora TU, Stein VM, O'Donnell P, Ruane T, Ward S, Crooks A, Li S, Mauldin E, Stellar S, De Meulder M, Kao ML, Ory DS, Davidson C, Vanier MT, Walkley SU. Vite CH, et al. Sci Transl Med. 2015 Feb 25;7(276):276ra26. doi: 10.1126/scitranslmed.3010101. Sci Transl Med. 2015. PMID: 25717099 Free PMC article.

10

Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates.

Hinderer C, Bell P, Louboutin JP, Zhu Y, Yu H, Lin G, Choa R, Gurda BL, Bagel J, O'Donnell P, Sikora T, Ruane T, Wang P, Tarantal AF, Casal ML, Haskins ME, Wilson JM. Hinderer C, et al. Mol Ther. 2015 Aug;23(8):1298-1307. doi: 10.1038/mt.2015.99. Epub 2015 May 29. Mol Ther. 2015. PMID: 26022732 Free PMC article.

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