Figure 1 | Gene Therapy

Figure 1

From: AAV9-mediated gene transfer of desmin ameliorates cardiomyopathy in desmin-deficient mice

Figure 1

Long-term desmin expression after intravenous injection of AAV-DES in DES-KO mice (n=10). (a) AAV-DES treatment resulted in desmin mRNA expression at levels of about 4.0±0.6% of those observed in wild-type mice (n=12). (b) Quantification of desmin protein expression revealed 22.2±9.8% of those in wild-type mice. (c) Detection of transgenic desmin expression in desmin-deficient mice (−/−, AAV-DES) in a representative western blot analysis (antibody DE-U-10). (d, e) Representative immunofluorescence staining of plasma membranes (green), nuclei (blue) and desmin (red, antibody DE-U-10) of an AAV-LUC-treated desmin-deficient mouse (n=10) (d), an AAV-DES-treated desmin-deficient mouse (n=10) (e) and a wild-type control (n=12) (f). Note that individual cardiomyocytes in AAV-DES-treated mice display variable levels of transgenic desmin protein expression with a cross-striated staining pattern and a signal accumulation at the level of intercalated discs.

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