@article{chen_genome_2021,
	title = {Genome editing using {CRISPR}/{Cas9} to treat hereditary hematological disorders.},
	doi = {10.1038/s41434-021-00247-9},
	abstract = {The clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9 (CRISPR/Cas9) system is a versatile and convenient genome-editing tool with prospects in gene therapy. This technique is based on customized site-specific nucleases with programmable guiding RNAs that cleave and introduce double-strand breaks (DSBs) at the target locus and achieve precise genome modification by triggering DNA repair mechanisms. Human hematopoietic stem/progenitor cells (HSPCs) are conventional cell targets for gene therapy in hematological diseases and have been widely used in most studies. Induced pluripotent stem cells (iPSCs) can be generated from a variety of somatic cells and hold great promise for personalized cell-based therapies. CRISPR/Cas9-mediated genome editing in autologous HSPCs and iPSCs is an ideal therapeutic solution for treating hereditary hematological disorders. Here, we review and summarize the latest studies about CRISPR/Cas9-mediated genome editing in patient-derived HSPCs and iPSCs to treat hereditary hematological disorders. Current challenges and prospects are also discussed.},
	journal = {Gene Therapy},
	author = {Chen, Yan and Wen, Ruiting and Yang, Zhigang and Chen, Zhanghui},
	year = {2021},
	doi = {10.1038/s41434-021-00247-9},
	note = {MAG ID: 3133509851},
	pages = {1--10},
}

{"_id":"Q5wcb3NMRptMgwp9A","bibbaseid":"chen-wen-yang-chen-genomeeditingusingcrisprcas9totreathereditaryhematologicaldisorders-2021","author_short":["Chen, Y.","Wen, R.","Yang, Z.","Chen, Z."],"bibdata":{"bibtype":"article","type":"article","title":"Genome editing using CRISPR/Cas9 to treat hereditary hematological disorders.","doi":"10.1038/s41434-021-00247-9","abstract":"The clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9 (CRISPR/Cas9) system is a versatile and convenient genome-editing tool with prospects in gene therapy. This technique is based on customized site-specific nucleases with programmable guiding RNAs that cleave and introduce double-strand breaks (DSBs) at the target locus and achieve precise genome modification by triggering DNA repair mechanisms. Human hematopoietic stem/progenitor cells (HSPCs) are conventional cell targets for gene therapy in hematological diseases and have been widely used in most studies. Induced pluripotent stem cells (iPSCs) can be generated from a variety of somatic cells and hold great promise for personalized cell-based therapies. CRISPR/Cas9-mediated genome editing in autologous HSPCs and iPSCs is an ideal therapeutic solution for treating hereditary hematological disorders. Here, we review and summarize the latest studies about CRISPR/Cas9-mediated genome editing in patient-derived HSPCs and iPSCs to treat hereditary hematological disorders. Current challenges and prospects are also discussed.","journal":"Gene Therapy","author":[{"propositions":[],"lastnames":["Chen"],"firstnames":["Yan"],"suffixes":[]},{"propositions":[],"lastnames":["Wen"],"firstnames":["Ruiting"],"suffixes":[]},{"propositions":[],"lastnames":["Yang"],"firstnames":["Zhigang"],"suffixes":[]},{"propositions":[],"lastnames":["Chen"],"firstnames":["Zhanghui"],"suffixes":[]}],"year":"2021","note":"MAG ID: 3133509851","pages":"1–10","bibtex":"@article{chen_genome_2021,\n\ttitle = {Genome editing using {CRISPR}/{Cas9} to treat hereditary hematological disorders.},\n\tdoi = {10.1038/s41434-021-00247-9},\n\tabstract = {The clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9 (CRISPR/Cas9) system is a versatile and convenient genome-editing tool with prospects in gene therapy. This technique is based on customized site-specific nucleases with programmable guiding RNAs that cleave and introduce double-strand breaks (DSBs) at the target locus and achieve precise genome modification by triggering DNA repair mechanisms. Human hematopoietic stem/progenitor cells (HSPCs) are conventional cell targets for gene therapy in hematological diseases and have been widely used in most studies. Induced pluripotent stem cells (iPSCs) can be generated from a variety of somatic cells and hold great promise for personalized cell-based therapies. CRISPR/Cas9-mediated genome editing in autologous HSPCs and iPSCs is an ideal therapeutic solution for treating hereditary hematological disorders. Here, we review and summarize the latest studies about CRISPR/Cas9-mediated genome editing in patient-derived HSPCs and iPSCs to treat hereditary hematological disorders. Current challenges and prospects are also discussed.},\n\tjournal = {Gene Therapy},\n\tauthor = {Chen, Yan and Wen, Ruiting and Yang, Zhigang and Chen, Zhanghui},\n\tyear = {2021},\n\tdoi = {10.1038/s41434-021-00247-9},\n\tnote = {MAG ID: 3133509851},\n\tpages = {1--10},\n}\n\n","author_short":["Chen, Y.","Wen, R.","Yang, Z.","Chen, Z."],"key":"chen_genome_2021","id":"chen_genome_2021","bibbaseid":"chen-wen-yang-chen-genomeeditingusingcrisprcas9totreathereditaryhematologicaldisorders-2021","role":"author","urls":{},"metadata":{"authorlinks":{}},"html":""},"bibtype":"article","biburl":"https://bibbase.org/zotero/kountour","dataSources":["MnayAXw3qciX87bz7"],"keywords":[],"search_terms":["genome","editing","using","crispr","cas9","treat","hereditary","hematological","disorders","chen","wen","yang","chen"],"title":"Genome editing using CRISPR/Cas9 to treat hereditary hematological disorders.","year":2021}