Delivering genes or RNAs to cells and tissues, or correcting existing genes, is an approach with great potential to treat a range of diseases. Since its inception about 20 years ago, the field of gene-based therapies has met with considerable challenges and controversy. However, recent progress has helped to realize the initial promise of these approaches and several gene-based therapies have now achieved success in clinical trials.
This Focus issue features four reviews from leading researchers to bring you up to date with progress in gene-based therapies, including gene delivery using viral vectors, strategies based on RNA-mediated silencing, and approaches that combine genetic manipulation with stem and progenitor cells.
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Research highlights
Getting RNAi therapies to the brain
Louisa Flintoft
doi:10.1038/nrg2990
Nature Reviews Genetics 12, 296 (2011)
Reviews
State-of-the-art gene-based therapies: the road ahead
Mark A. Kay
doi:10.1038/nrg2971
Nature Reviews Genetics 12, 316-328 (2011)
Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges
Federico Mingozzi & Katherine A. High
doi:10.1038/nrg2988
Nature Reviews Genetics 12, 341-355 (2011)
Ex vivo gene transfer and correction for cell-based therapies
Luigi Naldini
doi:10.1038/nrg2985
Nature Reviews Genetics 12, 301-315 (2011)
Current prospects for RNA interference-based therapies
Beverly L. Davidson & Paul B. McCray, Jr
doi:10.1038/nrg2968
Nature Reviews Genetics 12, 329-340 (2011)
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