Leone P - Search Results

1

Comparison of Endovascular and Intraventricular Gene Therapy With Adeno-Associated Virus-α-L-Iduronidase for Hurler Disease.

Janson CG, Romanova LG, Leone P, Nan Z, Belur L, McIvor RS, Low WC. Janson CG, et al. Among authors: leone p. Neurosurgery. 2014 Jan;74(1):99-111. doi: 10.1227/NEU.0000000000000157. Neurosurgery. 2014. PMID: 24077583 Free PMC article.

2

Long-term follow-up after gene therapy for canavan disease.

Leone P, Shera D, McPhee SW, Francis JS, Kolodny EH, Bilaniuk LT, Wang DJ, Assadi M, Goldfarb O, Goldman HW, Freese A, Young D, During MJ, Samulski RJ, Janson CG. Leone P, et al. Sci Transl Med. 2012 Dec 19;4(165):165ra163. doi: 10.1126/scitranslmed.3003454. Sci Transl Med. 2012. PMID: 23253610 Free PMC article.

3

Global CNS gene transfer for a childhood neurogenetic enzyme deficiency: Canavan disease.

Leone P, Janson CG, McPhee SJ, During MJ. Leone P, et al. Curr Opin Mol Ther. 1999 Aug;1(4):487-92. Curr Opin Mol Ther. 1999. PMID: 11713764 Review.

4

N-acetylaspartate supports the energetic demands of developmental myelination via oligodendroglial aspartoacylase.

Francis JS, Wojtas I, Markov V, Gray SJ, McCown TJ, Samulski RJ, Bilaniuk LT, Wang DJ, De Vivo DC, Janson CG, Leone P. Francis JS, et al. Among authors: leone p. Neurobiol Dis. 2016 Dec;96:323-334. doi: 10.1016/j.nbd.2016.10.001. Epub 2016 Oct 4. Neurobiol Dis. 2016. PMID: 27717881 Free PMC article.

5

Effects of AAV-2-mediated aspartoacylase gene transfer in the tremor rat model of Canavan disease.

McPhee SW, Francis J, Janson CG, Serikawa T, Hyland K, Ong EO, Raghavan SS, Freese A, Leone P. McPhee SW, et al. Among authors: leone p. Brain Res Mol Brain Res. 2005 Apr 27;135(1-2):112-21. doi: 10.1016/j.molbrainres.2004.12.007. Brain Res Mol Brain Res. 2005. PMID: 15857674

6

Lithium citrate for Canavan disease.

Janson CG, Assadi M, Francis J, Bilaniuk L, Shera D, Leone P. Janson CG, et al. Among authors: leone p. Pediatr Neurol. 2005 Oct;33(4):235-43. doi: 10.1016/j.pediatrneurol.2005.04.015. Pediatr Neurol. 2005. PMID: 16194720

7

Viral-based gene transfer to the mammalian CNS for functional genomic studies.

Janson CG, McPhee SW, Leone P, Freese A, During MJ. Janson CG, et al. Among authors: leone p. Trends Neurosci. 2001 Dec;24(12):706-12. doi: 10.1016/s0166-2236(00)01954-8. Trends Neurosci. 2001. PMID: 11718875 Review.

8

Immune responses to AAV in a phase I study for Canavan disease.

McPhee SW, Janson CG, Li C, Samulski RJ, Camp AS, Francis J, Shera D, Lioutermann L, Feely M, Freese A, Leone P. McPhee SW, et al. Among authors: leone p. J Gene Med. 2006 May;8(5):577-88. doi: 10.1002/jgm.885. J Gene Med. 2006. PMID: 16532510 Clinical Trial.

9

Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan disease.

Leone P, Janson CG, Bilaniuk L, Wang Z, Sorgi F, Huang L, Matalon R, Kaul R, Zeng Z, Freese A, McPhee SW, Mee E, During MJ. Leone P, et al. Ann Neurol. 2000 Jul;48(1):27-38. doi: 10.1002/1531-8249(200007)48:1<27::aid-ana6>3.0.co;2-6. Ann Neurol. 2000. PMID: 10894213 Clinical Trial.

10

Quantitative comparison of expression with adeno-associated virus (AAV-2) brain-specific gene cassettes.

Xu R, Janson CG, Mastakov M, Lawlor P, Young D, Mouravlev A, Fitzsimons H, Choi KL, Ma H, Dragunow M, Leone P, Chen Q, Dicker B, During MJ. Xu R, et al. Among authors: leone p. Gene Ther. 2001 Sep;8(17):1323-32. doi: 10.1038/sj.gt.3301529. Gene Ther. 2001. PMID: 11571569

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