دورية أكاديمية
Delivery of Genome Editing Reagents to Hematopoietic Stem/Progenitor Cells.
| العنوان: | Delivery of Genome Editing Reagents to Hematopoietic Stem/Progenitor Cells. |
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| المؤلفون: | Hoban MD; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, California., Romero Z; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, California., Cost GJ; Sangamo BioSciences, Richmond, California., Mendel M; Sangamo BioSciences, Richmond, California., Holmes M; Sangamo BioSciences, Richmond, California., Kohn DB; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, California.; Eli & Edythe Broad Center of Regenerative Medicine & Stem Cell Research, University of California, Los Angeles, Los Angeles, California. |
| المصدر: | Current protocols in stem cell biology [Curr Protoc Stem Cell Biol] 2016 Feb 03; Vol. 36, pp. 5B.4.1-5B.4.10. Date of Electronic Publication: 2016 Feb 03. |
| نوع المنشور: | Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't; Review |
| اللغة: | English |
| بيانات الدورية: | Country of Publication: United States NLM ID: 101470226 Publication Model: Electronic Cited Medium: Internet ISSN: 1938-8969 (Electronic) Linking ISSN: 19388969 NLM ISO Abbreviation: Curr Protoc Stem Cell Biol Subsets: MEDLINE |
| مواضيع طبية MeSH: | Genome, Human* , Hematopoietic Stem Cells*/cytology , Hematopoietic Stem Cells*/metabolism , Lentivirus*, Drug Delivery Systems/*methods , Electroporation/*methods , Transduction, Genetic/*methods, Animals ; Antigens, CD34 ; Humans |
| مستخلص: | This unit describes the protocol for the delivery of reagents for targeted genome editing to CD34(+) hematopoietic stem/progenitor cells (HSPCs). Specifically, this unit focuses on the process of thawing and pre-stimulating CD34(+) HSPCs, as well as the details of their electroporation with in vitro-transcribed mRNA-encoding site-specific nucleases [in this case zinc-finger nucleases (ZFNs)]. In addition, discussed is delivery of a gene editing donor template in the form of an oligonucleotide or integrase-defective lentiviral vector (IDLV). Finally, an analysis of cell survival following treatment and downstream culture conditions are presented. While optimization steps might be needed for each specific application with respect to nuclease and donor template amount, adherence to this protocol will serve as an excellent starting point for this further work. (Copyright © 2016 John Wiley & Sons, Inc.) |
| References: | Cartier, N., Hacein-Bey-Abina, S., Bartholomae, C.C., Veres, G., Schmidt, M., Kutschera, I., Vidaud, M., Abel, U., Dal-Cortivo, L., Caccavelli, L., Mahlaoui, N., Kiermer, V., Mittelstaedt, D., Bellesme, C., Lahlou, N., Lefrère, F., Blanche, S., Audit, M., Payen, E., Leboulch, P., l'Homme, B., Bougnères, P., Von Kalle, C., Fischer, A., Cavazzana-Calvo, M., and Aubourg, P. 2009. Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science (New York, N.Y.) 326:818-823. Available at: http://www.ncbi.nlm.nih.gov/pubmed/19892975 [Accessed March 27, 2015]. doi: 10.1126/science.1171242. Chapman, J.R., Taylor, M.R. G., and Boulton, S.J. 2012. Playing the end game: DNA double-strand break repair pathway choice. Mol. Cell 47:497-510. Available at: http://www.sciencedirect.com/science/article/pii/S1097276512006569 [Accessed July 14, 2014]. doi: 10.1016/j.molcel.2012.07.029. Choi, V.W., Asokan, A., Haberman, R.A., and Samulski, R.J. 2007. Production of recombinant adeno-associated viral vectors. Curr. Protoc. Hum. Genet Chapter 12:Unit 12.9. Available at: http://www.ncbi.nlm.nih.gov/pubmed/18428408 [Accessed October 14, 2015]. Cooper, A.R., Patel, S., Senadheera, S., Plath, K., Kohn, D.B., and Hollis, R.P. 2011. Highly efficient large-scale lentiviral vector concentration by tandem tangential flow filtration. J. Virol. Methods 177:1-9. Available at: http://www.pubmedcentral.nih.gov/articlerender.fcgi?artid=4276401&tool=pmcentrez&rendertype=abstract [Accessed March 24, 2015]. doi: 10.1016/j.jviromet.2011.06.019. Eichler, H., Beck, C., Bernard, F., Bugert, P., and Klüter, H. 2002. Use of recombinant human deoxyribonuclease (DNase) for processing of a thawed umbilical cord blood transplant in a patient with relapsed acute lymphoblastic leukemia. Ann. Hematol. 81:170-173. Available at: http://www.ncbi.nlm.nih.gov/pubmed/11904746 [Accessed August 13, 2015]. doi: 10.1007/s00277-001-0419-2. Gaj, T., Gersbach, C.A., and Barbas, C.F. 2013. ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering. Trends Biotechnol. 31:397-405. Available at: http://www.pubmedcentral.nih.gov/articlerender.fcgi?artid=3694601&tool=pmcentrez&rendertype=abstract [Accessed July 9, 2014]. doi: 10.1016/j.tibtech.2013.04.004. García-Piñeres, A.J., Hildesheim, A., Williams, M., Trivett, M., Strobl, S., and Pinto, L.A. 2006. DNase treatment following thawing of cryopreserved PBMC is a procedure suitable for lymphocyte functional studies. J. Immunol. Methods 313:209-213. Available at: http://www.ncbi.nlm.nih.gov/pubmed/16737707 [Accessed August 13, 2015]. doi: 10.1016/j.jim.2006.04.004. Genovese, P., Schiroli, G., Escobar, G., Di Tomaso, T., Firrito, C., Calabria, A., Moi, D., Mazzieri, R., Bonini, C., Holmes, M.C., Gregory, P.D., van der Burg, M., Gentner, B., Montini, E., Lombardo, A., and Naldini, L. 2014. Targeted genome editing in human repopulating haematopoietic stem cells. Nature 510:235-240. Available at: http://www.pubmedcentral.nih.gov/articlerender.fcgi?artid=4082311&tool=pmcentrez&rendertype=abstract [Accessed July 10, 2014]. doi: 10.1038/nature13420. Green, M. and Loewenstein, P.M. 2006. Human adenoviruses: Propagation, purification, quantification, and storage. Curr. Protoc. Microbiol. Chapter 14:Unit 14C.1. Available at: http://www.ncbi.nlm.nih.gov/pubmed/18770578 [Accessed October 26, 2015]. Hagedorn, E.J., Durand, E.M., Fast, E.M., and Zon, L.I. 2014. Getting more for your marrow: Boosting hematopoietic stem cell numbers with PGE2. Exp. Cell Res. 329:220-226. Available at: http://www.sciencedirect.com/science/article/pii/S0014482714003218 [Accessed July 5, 2015]. doi: 10.1016/j.yexcr.2014.07.030. Hendel, A., Bak, R.O., Clark, J.T., Kennedy, A.B., Ryan, D.E., Roy, S., Steinfeld, I., Lunstad, B.D., Kaiser, R.J., Wilkens, A.B., Bacchetta, R., Tsalenko, A., Dellinger, D., Bruhn, L., and Porteus, M.H. 2015. Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells. Nat. Biotechnol. Available at: http://www.ncbi.nlm.nih.gov/pubmed/26121415. Hendriks, W.T., Jiang, X., Daheron, L., and Cowan, C.A. 2015. TALEN- and CRISPR/Cas9-Mediated gene editing in human pluripotent stem cells using lipid-based transfection. Curr. Protoc. Stem Cell Biol. 34:5B.3.1-5B.3.25. Available at: http://www.ncbi.nlm.nih.gov/pubmed/26237572 [Accessed August 5, 2015]. Hoban, M.D., Cost, G.J., Mendel, M.C., Romero, Z., Kaufman, M.L., Joglekar, A. V, Ho, M., Lumaquin, D., Gray, D., Lill, G.R., Cooper, A.R., Urbinati, F., Senadheera, S., Zhu, A., Liu, P.Q., Paschon, D.E., Zhang, L., Rebar, E.J., Wilber, A., Wang, X., Gregory, P.D., Holmes, M.C., Reik, A., Hollis, R.P., and Kohn, D.B. 2015. Correction of the sickle-cell disease mutation in human hematopoietic stem/progenitor cells. Blood 125:2597-2604. Available at: http://www.bloodjournal.org/content/125/17/2597.abstract [Accessed March 4, 2015]. doi: 10.1182/blood-2014-12-615948. Holt, N., Wang, J., Kim, K., Friedman, G., Wang, X., Taupin, V., Crooks, G.M., Kohn, D.B., Gregory, P.D., Holmes, M.C., and Cannon, P.M. 2010. Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo. Nat. Biotechnol. 28:839-847. Available at: https://doi.org/10.1038/nbt.1663 [Accessed February 28, 2015]. doi: 10.1038/nbt.1663. Howes, R. and Schofield, C. 2015. Genome engineering using Adeno-Associated Virus (AAV). Methods Mol. Biol. 1239:75-103. Available at: http://www.ncbi.nlm.nih.gov/pubmed/25408402 [Accessed March 27, 2015]. doi: 10.1007/978-1-4939-1862-1_5. Logan, A.C., Lutzko, C., and Kohn, D.B. 2002. Advances in lentiviral vector design for gene-modification of hematopoietic stem cells. Curr. Opin. Biotechnol. 13:429-436. Available at: http://www.ncbi.nlm.nih.gov/pubmed/12459333 [Accessed March 27, 2015]. doi: 10.1016/S0958-1669(02)00346-4. Mandal, P.K., Ferreira, L.M. R., Collins, R., Meissner, T.B., Boutwell, C.L., Friesen, M., Vrbanac, V., Garrison, B.S., Stortchevoi, A., Bryder, D., Musunuru, K., Brand, H., Tager, A.M., Allen, T.M., Talkowski, M.E., Rossi, D.J., and Cowan, C.A. 2014. Efficient ablation of genes in human hematopoietic stem and effector cells using CRISPR/Cas9. Cell Stem Cell 15:643-652. Available at: http://www.ncbi.nlm.nih.gov/pubmed/25517468 [Accessed November 7, 2014]. doi: 10.1016/j.stem.2014.10.004. Nightingale, S.J., Hollis, R.P., Pepper, K.A., Petersen, D., Yu, X.-J., Yang, C., Bahner, I., and Kohn, D.B. 2006. Transient gene expression by nonintegrating lentiviral vectors. Mol. Ther. 13:1121-1132. Available at: http://www.ncbi.nlm.nih.gov/pubmed/16556511 [Accessed February 20, 2015]. doi: 10.1016/j.ymthe.2006.01.008. Phelan, M.C. 2006. Techniques for mammalian cell tissue culture. Curr. Opin. Biotechnol. Appendix 3:Appendix 3B. Available at: http://www.ncbi.nlm.nih.gov/pubmed/18770828 [Accessed July 15, 2015]. Shayakhmetov, D.M., Carlson, C.A., Stecher, H., Li, Q., Stamatoyannopoulos, G., and Lieber, A. 2002. A high-capacity, capsid-modified hybrid adenovirus/adeno-associated virus vector for stable transduction of human hematopoietic cells. J. Virol. 76:1135-1143. Available at: http://www.pubmedcentral.nih.gov/articlerender.fcgi?artid=135810&tool=pmcentrez&rendertype=abstract [Accessed March 24, 2015]. doi: 10.1128/JVI.76.3.1135-1143.2002. Van Driessche, A., Ponsaerts, P., Van Bockstaele, D.R., Van Tendeloo, V.F.I., and Berneman, Z.N. 2005. Messenger RNA electroporation: An efficient tool in immunotherapy and stem cell research. Folia Histochem. Cytobiol. 43:213-216. Available at: http://www.ncbi.nlm.nih.gov/pubmed/16382887 [Accessed March 24, 2015]. |
| معلومات مُعتمدة: | 5 T32 AI060567 United States AI NIAID NIH HHS |
| فهرسة مساهمة: | Keywords: electroporation; genome editing; hematopoietic stem cells; targeted nucleases |
| المشرفين على المادة: | 0 (Antigens, CD34) |
| تواريخ الأحداث: | Date Created: 20160204 Date Completed: 20161027 Latest Revision: 20200108 |
| رمز التحديث: | 20231215 |
| DOI: | 10.1002/9780470151808.sc05b04s36 |
| PMID: | 26840227 |
| قاعدة البيانات: | MEDLINE |
| تدمد: | 1938-8969 |
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| DOI: | 10.1002/9780470151808.sc05b04s36 |