State-of-the-art gene-based therapies: the road ahead

Author:

Kay Mark A.

Publisher

Springer Science and Business Media LLC

Subject

Genetics(clinical),Genetics,Molecular Biology

Reference125 articles.

1. Mingozzi, F. & High, K. A. Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges. Nature Rev. Genet. (in the press).

2. Davidson, B. L. & McCray, P. B. Jr. Current prospects for RNA interference-based therapies. Nature Rev. Genet. (in the press).

3. Naldini, L. Ex vivo gene transfer and correction for cell-based therapies. Nature Rev. Genet. (in the press).

4. Urnov, F. D., Rebar, E. J., Holmes, M. C., Zhang, H. S. & Gregory, P. D. Genome editing with engineered zinc finger nucleases. Nature Rev. Genet. 11, 636–646 (2010).

5. Lu, Q. L. et al. The status of exon skipping as a therapeutic approach to duchenne muscular dystrophy. Mol. Ther. 19, 9–15 (2011).

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