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Correction of the enzymatic and functional deficits in a model of Pompe disease using adeno-associated virus vectors.
Mol Ther. 2002 May;5(5 Pt 1):571-8. doi: 10.1006/mthe.2002.0580.
Mol Ther. 2002.
PMID: 11991748
Free article.
Dual vectors expressing murine factor VIII result in sustained correction of hemophilia A mice.
Mah C, Sarkar R, Zolotukhin I, Schleissing M, Xiao X, Kazazian HH, Byrne BJ.
Mah C, et al.
Hum Gene Ther. 2003 Jan 20;14(2):143-52. doi: 10.1089/104303403321070838.
Hum Gene Ther. 2003.
PMID: 12614565
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