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Items: 15


Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-associated virus clinical trial.

Monahan PE, Sun J, Gui T, Hu G, Hannah WB, Wichlan DG, Wu Z, Grieger JC, Li C, Suwanmanee T, Stafford DW, Booth CJ, Samulski JJ, Kafri T, McPhee SW, Samulski RJ.

Hum Gene Ther. 2015 Feb;26(2):69-81. doi: 10.1089/hum.2014.106. Epub 2015 Jan 21.


Efficient transduction of vascular smooth muscle cells with a translational AAV2.5 vector: a new perspective for in-stent restenosis gene therapy.

Lompré AM, Hadri L, Merlet E, Keuylian Z, Mougenot N, Karakikes I, Chen J, Atassi F, Marchand A, Blaise R, Limon I, McPhee SW, Samulski RJ, Hajjar RJ, Lipskaia L.

Gene Ther. 2013 Sep;20(9):901-12. doi: 10.1038/gt.2013.13. Epub 2013 Mar 28.


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.

Sci Transl Med. 2012 Dec 19;4(165):165ra163. doi: 10.1126/scitranslmed.3003454.


Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector.

Bowles DE, McPhee SW, Li C, Gray SJ, Samulski JJ, Camp AS, Li J, Wang B, Monahan PE, Rabinowitz JE, Grieger JC, Govindasamy L, Agbandje-McKenna M, Xiao X, Samulski RJ.

Mol Ther. 2012 Feb;20(2):443-55. doi: 10.1038/mt.2011.237. Epub 2011 Nov 8.


Assessment of hippocampal adeno-associated viral vector gene delivery via frameless stereotaxis in a nonhuman primate.

Leung CH, Kliem MA, Heeke BL, McPhee SW, Federici T, Snyder BR, Boulis NM.

Stereotact Funct Neurosurg. 2011;89(5):275-85. doi: 10.1159/000328265. Epub 2011 Aug 18.


Dystrophin immunity in Duchenne's muscular dystrophy.

Mendell JR, Campbell K, Rodino-Klapac L, Sahenk Z, Shilling C, Lewis S, Bowles D, Gray S, Li C, Galloway G, Malik V, Coley B, Clark KR, Li J, Xiao X, Samulski J, McPhee SW, Samulski RJ, Walker CM.

N Engl J Med. 2010 Oct 7;363(15):1429-37. doi: 10.1056/NEJMoa1000228.


Adeno-associated virus for the treatment of muscle diseases: toward clinical trials.

DiPrimio N, McPhee SW, Samulski RJ.

Curr Opin Mol Ther. 2010 Oct;12(5):553-60. Review.


Gene therapy for cardiomyocytes, a heart beat away.

McPhee SW, Samulski RJ.

Gene Ther. 2009 Jun;16(6):707-8. doi: 10.1038/gt.2009.40. Epub 2009 Apr 30. No abstract available.


Natural history of Canavan disease revealed by proton magnetic resonance spectroscopy (1H-MRS) and diffusion-weighted MRI.

Janson CG, McPhee SW, Francis J, Shera D, Assadi M, Freese A, Hurh P, Haselgrove J, Wang DJ, Bilaniuk L, Leone P.

Neuropediatrics. 2006 Aug;37(4):209-21.


Novel role for aspartoacylase in regulation of BDNF and timing of postnatal oligodendrogenesis.

Francis JS, Olariu A, McPhee SW, Leone P.

J Neurosci Res. 2006 Jul;84(1):151-69.


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.

J Gene Med. 2006 May;8(5):577-88.


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.

Brain Res Mol Brain Res. 2005 Apr 27;135(1-2):112-21.


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

Janson CG, McPhee SW, Leone P, Freese A, During MJ.

Trends Neurosci. 2001 Dec;24(12):706-12. Review. Erratum in: Trends Neurosci 2002 Jan;25(1):59.


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.

Ann Neurol. 2000 Jul;48(1):27-38. Erratum in: Ann Neurol 2000 Sep;48(3):398. Bilianuk L [corrected to Bilaniuk L].


Multi-site partitioned delivery of human tyrosine hydroxylase gene with phenotypic recovery in Parkinsonian rats.

Leone P, McPhee SW, Janson CG, Davidson BL, Freese A, During MJ.

Neuroreport. 2000 Apr 27;11(6):1145-51.


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