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[CRISPR/Cas: technique to repair DNA errors: is a clinical breakthrough near?]
Ned Tijdschr Geneeskd. 2018 Jun 29; 162NT

Abstract

CRISPR/Cas gene editing makes it much easier to make targeted changes in the DNA of human cells than other forms of gene therapy. This revolutionary technology offers spectacular opportunities to study gene functions; the clinical consequences of gene variations in patients can be determined much faster. The efficacy and accuracy of CRISPR/Cas is so impressive that a breakthrough to therapeutic applications is approaching fast. CRISPR/Cas is already being used in immunotherapy against cancer, and trials for monogenetic blood disorders, such as beta-thalassemia, have been scheduled. However, broad clinical implementation of CRISPR/Cas is not feasible yet, due to off-target DNA changes that may occur as a by-product. Particularly in case of in-vivo applications there are therapeutic challenges. For gene editing in human embryos, technical shortcomings and open ethical issues need to be addressed. Gene-editing therapy for serious disorders with transplantable cell types, and therefore the option of verification of "CRISPRed" cells, is seen as a possible first application within the regular healthcare system.

Authors+Show Affiliations

Amsterdam Cancer Center, Amsterdam UMC, afd. Klinische Genetica, sectie Oncogenetica, Amsterdam. Contact: H.J. van de Vrugt (h.vandevrugt@vumc.nl).Amsterdam Cancer Center, Amsterdam UMC, afd. Klinische Genetica, sectie Community Genetics, Amsterdam.Amsterdam Cancer Center, Amsterdam UMC, afd. Klinische Genetica, sectie Oncogenetica, Amsterdam.

Pub Type(s)

Journal Article

Language

dut

PubMed ID

30040285

Citation

van de Vrugt, Henri J., et al. "[CRISPR/Cas: Technique to Repair DNA Errors: Is a Clinical Breakthrough Near?]." Nederlands Tijdschrift Voor Geneeskunde, vol. 162, 2018.
van de Vrugt HJ, Cornel MC, Wolthuis RMF. [CRISPR/Cas: technique to repair DNA errors: is a clinical breakthrough near?]. Ned Tijdschr Geneeskd. 2018;162.
van de Vrugt, H. J., Cornel, M. C., & Wolthuis, R. M. F. (2018). [CRISPR/Cas: technique to repair DNA errors: is a clinical breakthrough near?]. Nederlands Tijdschrift Voor Geneeskunde, 162.
van de Vrugt HJ, Cornel MC, Wolthuis RMF. [CRISPR/Cas: Technique to Repair DNA Errors: Is a Clinical Breakthrough Near?]. Ned Tijdschr Geneeskd. 2018 Jun 29;162 PubMed PMID: 30040285.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - [CRISPR/Cas: technique to repair DNA errors: is a clinical breakthrough near?] AU - van de Vrugt,Henri J, AU - Cornel,Martina C, AU - Wolthuis,Rob M F, Y1 - 2018/06/29/ PY - 2018/7/25/entrez PY - 2018/7/25/pubmed PY - 2018/12/21/medline JF - Nederlands tijdschrift voor geneeskunde JO - Ned Tijdschr Geneeskd VL - 162 N2 - CRISPR/Cas gene editing makes it much easier to make targeted changes in the DNA of human cells than other forms of gene therapy. This revolutionary technology offers spectacular opportunities to study gene functions; the clinical consequences of gene variations in patients can be determined much faster. The efficacy and accuracy of CRISPR/Cas is so impressive that a breakthrough to therapeutic applications is approaching fast. CRISPR/Cas is already being used in immunotherapy against cancer, and trials for monogenetic blood disorders, such as beta-thalassemia, have been scheduled. However, broad clinical implementation of CRISPR/Cas is not feasible yet, due to off-target DNA changes that may occur as a by-product. Particularly in case of in-vivo applications there are therapeutic challenges. For gene editing in human embryos, technical shortcomings and open ethical issues need to be addressed. Gene-editing therapy for serious disorders with transplantable cell types, and therefore the option of verification of "CRISPRed" cells, is seen as a possible first application within the regular healthcare system. SN - 1876-8784 UR - https://www.unboundmedicine.com/medline/citation/30040285/[CRISPR/Cas:_technique_to_repair_DNA_errors:_is_a_clinical_breakthrough_near] L2 - https://medlineplus.gov/genesandgenetherapy.html DB - PRIME DP - Unbound Medicine ER -