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Moving towards a cu...
Moving towards a cure in genetics : what is needed to bring somatic gene therapy to the clinic?
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- Cornel, Martina C. (författare)
- Vrije Univ Amsterdam, Amsterdam UMC, Clin Genet & Amsterdam Publ Hlth Res Inst, Sect Community Genet, Boelelaan 1117, Amsterdam, Netherlands
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- Howard, Heidi Carmen (författare)
- Uppsala universitet,Centrum för forsknings- och bioetik
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- Lim, Daniel (författare)
- Kirkland & Ellis Int LLP, London, England
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- Bonham, Vence L. (författare)
- NHGRI, Social & Behav Res Branch, NIH, Bethesda, MD 20892 USA
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- Wartiovaara, Kirmo (författare)
- Univ Helsinki, Helsinki Univ Hosp, Clin Genet, Meilahdentie 2, Helsinki 00290, Finland
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(creator_code:org_t)
- 2018-12-19
- 2019
- Engelska.
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Ingår i: European Journal of Human Genetics. - : NATURE PUBLISHING GROUP. - 1018-4813 .- 1476-5438. ; 27:3, s. 484-487
- Relaterad länk:
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https://www.nature.c...
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https://urn.kb.se/re...
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https://doi.org/10.1...
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Abstract
Ämnesord
Stäng
- 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.
Ämnesord
- MEDICIN OCH HÄLSOVETENSKAP -- Medicinska och farmaceutiska grundvetenskaper -- Medicinsk genetik (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Basic Medicine -- Medical Genetics (hsv//eng)
Publikations- och innehållstyp
- ref (ämneskategori)
- art (ämneskategori)
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