Molecular Therapy

FIGURE 1

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Helper-Independent and AAV-ITR-Independent Chromosomal Integration of Double-Stranded Linear DNA Vectors in Mice

Hiroyuki Nakai, Eugenio Montini, Sally Fuess, Theresa A. Storm, Leonard Meuse, Milton Finegold, Markus Grompe and Mark A. Kay

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Figure 1.

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Restriction enzyme maps and schematic representation of circular and linear ds DNA vectors. (A–C) Maps of hFAH or hF.IX expression cassettes used in the ds DNA vectors. Locations of restriction enzyme recognition sites and probes used for Southern blot analysis are indicated. Asterisks represent DpnI recognition sites. (D) Structures of L+, L-, C+, and C- vectors. All the hFAH-expressing ds DNA vectors and the AAV-hFAH vector carried cassette (A), hF.IX-C+ and hF.IX2-C- had cassette (B), and hF.IX-L+ and L- carried cassette (C). deltaEF1alphaEP13 and deltaDEF1alphaEP26 are two truncated versions of the EF1alpha gene enhancer–promoter. Abbreviations for restriction enzymes: D, DraI; B, BamHI; N, NsiI; Bg, BglII; S, SacI.

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