A MicroRNA124 Target Sequence Restores Astrocyte Specificity of gfaABC1D-Driven Transgene Expression in AAV-Mediated Gene Transfer

Grit Taschenberger, Julia Tereshchenko, Sebastian Kügler

Molecular Therapy — Nucleic Acids · 2017 · 51 citations · 39 references

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Abstract

D promoter on its own is not sufficient to specifically target transgene expression to astrocytes and is not well suited for AAV-based gene targeting, even if short promoter sequences are required. The combination with a miR de-targeting sequence represents a promising experimental strategy that eliminates off-target, neuronal expression.

References

39