Key result
In vivo delta-sarcoglycan gene transfer drives expression of all four sarcoglycans in hamster hearts.
In vivo delta-sarcoglycan gene transfer successfully restores the expression of the entire sarcoglycan complex in a hamster model of dilated cardiomyopathy.
May support sarcoglycan gene therapy development in cardiomyopathy models; leaves open translation to human disease.
The delta-sarcoglycan (SG) gene is deleted in hamsters with hereditary cardiomyopathies. Immunological analyses of heart before, but not after, the progression of cardiomyopathy (CM) revealed that the BIO 14.6 strain, a model of hypertrophic CM, heterogeneously preserved alpha- and gamma-SG with loss of beta- and delta-SG. In contrast, the TO-2 strain, a model of dilated CM, did not show either SG. Furthermore, in vivo transfer of the full length delta-SG gene to TO-2 hearts expressed all four SGs. Thus, this age- and strain-dependent features suggest a more feasible setting for TO-2 than BIO 14.6 to verify both CM progression and the efficacy of gene therapy.
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Kawada et al. (1999) studied Hereditary cardiomyopathies. In vivo transfer of the full length delta-sarcoglycan gene was evaluated on Expression of sarcoglycans. In vivo transfer of the full-length delta-sarcoglycan gene to TO-2 hamster hearts resulted in the expression of all four sarcoglycans.
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