Moving towards a cure in genetics: what is needed to bring somatic gene therapy to the clinic?
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Updated: Jan 31, 2026

Detecting Somatic Genetic Alterations in Tumor Specimens by Exon Capture and Massively Parallel Sequencing
Published on: October 18, 2013
Martina C Cornel1, Heidi C Howard2, Daniel Lim3
1Amsterdam UMC, Clinical Genetics and Amsterdam Public Health Research Institute, Section Community Genetics, Vrije Universiteit Amsterdam, De Boelelaan 1117, Amsterdam, The Netherlands. mc.cornel@vumc.nl.
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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