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Gene knock-outs in human CD34+ hematopoietic stem and progenitor cells and in the human immune system of mice.

التفاصيل البيبلوغرافية
العنوان: Gene knock-outs in human CD34+ hematopoietic stem and progenitor cells and in the human immune system of mice.
المؤلفون: Daniel A Kuppers, Jonathan Linton, Sergio Ortiz Espinosa, Kelly M McKenna, Anthony Rongvaux, Patrick J Paddison
المصدر: PLoS ONE, Vol 18, Iss 6, p e0287052 (2023)
بيانات النشر: Public Library of Science (PLoS), 2023.
سنة النشر: 2023
المجموعة: LCC:Medicine
LCC:Science
مصطلحات موضوعية: Medicine, Science
الوصف: Human CD34+ hematopoietic stem and progenitor cells (HSPCs) are a standard source of cells for clinical HSC transplantations as well as experimental xenotransplantation to generate "humanized mice". To further extend the range of applications of these humanized mice, we developed a protocol to efficiently edit the genomes of human CD34+ HSPCs before transplantation. In the past, manipulating HSPCs has been complicated by the fact that they are inherently difficult to transduce with lentivectors, and rapidly lose their stemness and engraftment potential during in vitro culture. However, with optimized nucleofection of sgRNA:Cas9 ribonucleoprotein complexes, we are now able to edit a candidate gene in CD34+ HSPCs with almost 100% efficiency, and transplant these modified cells in immunodeficient mice with high engraftment levels and multilineage hematopoietic differentiation. The result is a humanized mouse from which we knocked out a gene of interest from their human immune system.
نوع الوثيقة: article
وصف الملف: electronic resource
اللغة: English
تدمد: 1932-6203
Relation: https://doaj.org/toc/1932-6203
DOI: 10.1371/journal.pone.0287052
URL الوصول: https://doaj.org/article/c40eaaf9412747dea3045397d9b776a9
رقم الأكسشن: edsdoj.40eaaf9412747dea3045397d9b776a9
قاعدة البيانات: Directory of Open Access Journals
الوصف
تدمد:19326203
DOI:10.1371/journal.pone.0287052