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. 2019 Mar;27(3):484-487.
doi: 10.1038/s41431-018-0309-x. Epub 2018 Dec 19.

Moving towards a cure in genetics: what is needed to bring somatic gene therapy to the clinic?

Martina C Cornel  1 , Heidi C Howard  2 , Daniel Lim  3 , Vence L Bonham  4 , Kirmo Wartiovaara  5
Affiliations

Moving towards a cure in genetics: what is needed to bring somatic gene therapy to the clinic?

Martina C Cornel et al. Eur J Hum Genet. 2019 Mar.

Abstract

Clinical trials using somatic gene editing (e.g., CRISPR-Cas9) have started in Europe and the United States and may provide safe and effective treatment and cure, not only for cancers but also for some monogenic conditions. In a workshop at the 2018 European Human Genetics Conference, the challenges of bringing somatic gene editing therapies to the clinic were discussed. The regulatory process needs to be considered early in the clinical development pathway to produce the data necessary to support the approval by the European Medicines Agency. The roles and responsibilities for geneticists may include counselling to explain the treatment possibilities and safety interpretation.

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Conflict of interest statement

The authors declare that they have no conflict of interest.

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