دورية أكاديمية
أعرض في EDS

Technical considerations for the use of CRISPR/Cas9 in hematology research.

التفاصيل البيبلوغرافية
العنوان: Technical considerations for the use of CRISPR/Cas9 in hematology research.
المؤلفون: Gundry MC; Department of Molecular and Human Genetics, Stem Cells and Regenerative Medicine Center, Center for Cell and Gene Therapy, Baylor College of Medicine, Houston, TX., Dever DP; Department of Pediatrics, Stanford University, Stanford, CA., Yudovich D; Division of Molecular Medicine and Gene Therapy, Lund Stem Cell Center, Lund University, Lund, Sweden., Bauer DE; Division of Hematology/Oncology, Boston Children's Hospital, Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Stem Cell Institute, Department of Pediatrics, Harvard Medical School, Boston, MA., Haas S; Heidelberg Institute for Stem Cell Technology and Experimental Medicine and Division of Stem Cells and Cancer, DKFZ German Cancer Research Centre, Heidelberg, Germany., Wilkinson AC; Stanford Institute for Stem Cell Biology and Regenerative Medicine, Stanford University, Stanford, CA. Electronic address: adamcw@stanford.edu., Singbrant S; Division of Molecular Medicine and Gene Therapy, Lund Stem Cell Center, Lund University, Lund, Sweden.
المصدر: Experimental hematology [Exp Hematol] 2017 Oct; Vol. 54, pp. 4-11. Date of Electronic Publication: 2017 Jul 27.
نوع المنشور: Journal Article; Review; Research Support, Non-U.S. Gov't; Research Support, N.I.H., Extramural
اللغة: English
بيانات الدورية: Publisher: Elsevier Science Inc Country of Publication: Netherlands NLM ID: 0402313 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1873-2399 (Electronic) Linking ISSN: 0301472X NLM ISO Abbreviation: Exp Hematol Subsets: MEDLINE
أسماء مطبوعة: Publication: 1999- : Amsterdam : Elsevier Science Inc.
Original Publication: Copenhagen, Munksgaard.
مواضيع طبية MeSH: CRISPR-Cas Systems* , Genome* , Mutagenesis*, Gene Editing/*methods , Gene Targeting/*methods , Hematology/*methods, Animals ; Computational Biology ; Genetic Vectors/chemistry ; Genetic Vectors/metabolism ; Hematopoiesis/genetics ; Hematopoietic Stem Cell Transplantation ; Hematopoietic Stem Cells/cytology ; Hematopoietic Stem Cells/metabolism ; Humans ; Lentivirus/genetics ; Lentivirus/metabolism ; Multipotent Stem Cells/cytology ; Multipotent Stem Cells/metabolism ; RNA, Guide, CRISPR-Cas Systems/genetics ; RNA, Guide, CRISPR-Cas Systems/metabolism
مستخلص: The hematopoietic system is responsible for transporting oxygen and nutrients, fighting infections, and repairing tissue damage. Hematopoietic system dysfunction therefore causes a range of serious health consequences. Lifelong hematopoiesis is maintained by repopulating multipotent hematopoietic stem cells (HSCs) that replenish shorter-lived, mature blood cell types. A prokaryotic mechanism of immunity, the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9 nuclease system, has been recently "repurposed" to mutate mammalian genomes efficiently and in a sequence-specific manner. The application of this genome-editing technology to hematology has afforded new approaches for functional genomics and even the prospect of "correcting" dysfunctional HSCs in the treatment of serious genetic hematological diseases. In this Perspective, we provide an overview of three recent CRISPR/Cas9 methods in hematology: gene disruption, gene targeting, and saturating mutagenesis. We also summarize the technical considerations and advice provided during the May 2017 International Society of Experimental Hematology New Investigator Committee webinar on the same topic.
(Copyright © 2017 ISEH – Society for Hematology and Stem Cells. Published by Elsevier Inc. All rights reserved.)
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معلومات مُعتمدة: DP2 HL137300 United States HL NHLBI NIH HHS; K08 DK093705 United States DK NIDDK NIH HHS; R03 DK109232 United States DK NIDDK NIH HHS; UL1 TR001085 United States TR NCATS NIH HHS
المشرفين على المادة: 0 (RNA, Guide, CRISPR-Cas Systems)
تواريخ الأحداث: Date Created: 20170801 Date Completed: 20171004 Latest Revision: 20240104
رمز التحديث: 20240104
مُعرف محوري في PubMed: PMC5603407
DOI: 10.1016/j.exphem.2017.07.006
PMID: 28757433
قاعدة البيانات: MEDLINE
الوصف
تدمد:1873-2399
DOI:10.1016/j.exphem.2017.07.006