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Updated: Jul 10, 2026

08:54
In vivo Application of the REMOTE-control System for the Manipulation of Endogenous Gene Expression
Published on: March 29, 2019
[Gene therapy in The Netherlands].
E A M Schenk-Braat1, M M K B van Mierlo, G A P Hospers
1Erasmus MC-Centrum, Postbus 2040, 3000 CA Rotterdam. e.schenk-braat@erasmusmc.nl
Nederlands Tijdschrift Voor Geneeskunde
|October 24, 2007
Summary
Gene therapy shows promise for genetic disorders like severe combined immune deficiency and haemophilia B, with ongoing global research. While generally safe, its effectiveness for cancer treatment remains limited.
Area of Science:
- Biomedical research
- Clinical trials
- Gene therapy
Context:
- Global research on gene therapy's clinical utility is extensive.
- Two gene therapy products recently approved in China.
- Over 200 Dutch patients treated in gene therapy clinical trials.
Purpose:
- To review the current status and clinical utility of gene therapy.
- To assess the safety and efficacy of gene therapy for various conditions.
Summary:
- Gene therapy is generally safe based on published results.
- Effective for certain genetic disorders like severe combined immune deficiency and haemophilia B.
- Efficacy in cancer treatment appears limited currently.
Impact:
- Gene therapy offers potential for treating genetic disorders.
- Further research is needed to improve cancer treatment efficacy.
- Accumulating clinical experience informs future gene therapy applications.
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