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Efficient non-viral immune cell engineering using circular single-stranded DNA-mediated genomic integration 利用环状单链dna介导的基因组整合进行高效的非病毒免疫细胞工程
IF 46.9 1区 生物学
Nature biotechnology Pub Date : 2024-12-11 DOI: 10.1038/s41587-024-02504-9
Keqiang Xie, Jakob Starzyk, Ishita Majumdar, Jiao Wang, Katerina Rincones, Thao Tran, Danna Lee, Sarah Niemi, John Famiglietti, Bernhard Suter, Richard Shan, Hao Wu
{"title":"Efficient non-viral immune cell engineering using circular single-stranded DNA-mediated genomic integration","authors":"Keqiang Xie, Jakob Starzyk, Ishita Majumdar, Jiao Wang, Katerina Rincones, Thao Tran, Danna Lee, Sarah Niemi, John Famiglietti, Bernhard Suter, Richard Shan, Hao Wu","doi":"10.1038/s41587-024-02504-9","DOIUrl":"https://doi.org/10.1038/s41587-024-02504-9","url":null,"abstract":"<p>The use of adeno-associated viruses (AAVs) as donors for homology-directed repair (HDR)-mediated genome engineering is limited by safety issues, manufacturing constraints and restricted packaging limits. Non-viral targeted genetic knock-ins rely primarily on double-stranded DNA (dsDNA) and linear single-stranded DNA (lssDNA) donors. dsDNA is known to have low efficiency and high cytotoxicity, while lssDNA is challenging for scaled manufacture. In this study, we developed a non-viral genome writing catalyst (GATALYST) system that allows production of circular single-stranded DNAs (cssDNAs) up to approximately 20 kilobases as donor templates for highly efficient precision transgene integration. cssDNA donors enable knock-in efficiency of up to 70% in induced pluripotent stem cells (iPSCs) and improved efficiency in multiple clinically relevant primary immune cell types and at multiple genomic loci implicated for clinical applications with various nuclease editor systems. The high precision and efficiency in chimeric antigen receptor (CAR)-T and natural killer (NK) cells, improved safety, payload flexibility and scalable manufacturability of cssDNA shows potential for future applications of genome engineering.</p>","PeriodicalId":19084,"journal":{"name":"Nature biotechnology","volume":"25 6 1","pages":""},"PeriodicalIF":46.9,"publicationDate":"2024-12-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142805096","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
A technical approach to global plant genome editing regulation 全球植物基因组编辑调控的技术途径
IF 33.1 1区 生物学
Nature biotechnology Pub Date : 2024-12-11 DOI: 10.1038/s41587-024-02489-5
Evan Groover, Elizabeth Njuguna, Kailash Chander Bansal, Anne Muia, Musa Kwehangana, Christopher Simuntala, Richard Lloyd Mills, Emmanuel Kwakye, Pedro Rocha, Josephine Amedu, Eduardo Morillo, Mohana Anita Anthonysamy, A. B. M. Khaldun, Lilian Chimpepo, Massouroudini Akoudjin, D. M. J. B. Senanayake, Dechen Wangmo, Dessalegn Atnafu, Geronima P. Eusebio, Chalinee Kongsawat, Melinda Kliegman
{"title":"A technical approach to global plant genome editing regulation","authors":"Evan Groover,&nbsp;Elizabeth Njuguna,&nbsp;Kailash Chander Bansal,&nbsp;Anne Muia,&nbsp;Musa Kwehangana,&nbsp;Christopher Simuntala,&nbsp;Richard Lloyd Mills,&nbsp;Emmanuel Kwakye,&nbsp;Pedro Rocha,&nbsp;Josephine Amedu,&nbsp;Eduardo Morillo,&nbsp;Mohana Anita Anthonysamy,&nbsp;A. B. M. Khaldun,&nbsp;Lilian Chimpepo,&nbsp;Massouroudini Akoudjin,&nbsp;D. M. J. B. Senanayake,&nbsp;Dechen Wangmo,&nbsp;Dessalegn Atnafu,&nbsp;Geronima P. Eusebio,&nbsp;Chalinee Kongsawat,&nbsp;Melinda Kliegman","doi":"10.1038/s41587-024-02489-5","DOIUrl":"10.1038/s41587-024-02489-5","url":null,"abstract":"The Innovate Genomics Institute brought together regulators from 16 countries to discuss global capacity building for the regulation of genome-edited crops. The workshop provided insights into the suitable use of technical analyses to validate edits and raised future considerations regarding regulation reporting, offering suggestions to help countries meet their objectives in the ever-growing landscape of genome editing techniques.","PeriodicalId":19084,"journal":{"name":"Nature biotechnology","volume":"42 12","pages":"1773-1780"},"PeriodicalIF":33.1,"publicationDate":"2024-12-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142809378","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Intellectual property training should be embedded in the biomedical education process 知识产权培训应纳入生物医学教育过程
IF 33.1 1区 生物学
Nature biotechnology Pub Date : 2024-12-11 DOI: 10.1038/s41587-024-02499-3
Randal A. Serafini, Micaila D. E. Curtis, Stella Alimperti
{"title":"Intellectual property training should be embedded in the biomedical education process","authors":"Randal A. Serafini,&nbsp;Micaila D. E. Curtis,&nbsp;Stella Alimperti","doi":"10.1038/s41587-024-02499-3","DOIUrl":"10.1038/s41587-024-02499-3","url":null,"abstract":"Implementation of intellectual property education in academic institutions can result in increased opportunities for protecting intellectual property and limit costs, but to be successful technology transfer offices must also adapt.","PeriodicalId":19084,"journal":{"name":"Nature biotechnology","volume":"42 12","pages":"1924-1925"},"PeriodicalIF":33.1,"publicationDate":"2024-12-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.com/articles/s41587-024-02499-3.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142809220","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Base editing boosts hemoglobin in sickle cell disease 碱基编辑增加镰状细胞病中的血红蛋白
IF 33.1 1区 生物学
Nature biotechnology Pub Date : 2024-12-11 DOI: 10.1038/s41587-024-02517-4
{"title":"Base editing boosts hemoglobin in sickle cell disease","authors":"","doi":"10.1038/s41587-024-02517-4","DOIUrl":"10.1038/s41587-024-02517-4","url":null,"abstract":"","PeriodicalId":19084,"journal":{"name":"Nature biotechnology","volume":"42 12","pages":"1759-1759"},"PeriodicalIF":33.1,"publicationDate":"2024-12-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142809417","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Discovering the crucial function of long noncoding RNAs 发现长链非编码rna的关键功能
IF 33.1 1区 生物学
Nature biotechnology Pub Date : 2024-12-11 DOI: 10.1038/s41587-024-02509-4
Iris Marchal
{"title":"Discovering the crucial function of long noncoding RNAs","authors":"Iris Marchal","doi":"10.1038/s41587-024-02509-4","DOIUrl":"10.1038/s41587-024-02509-4","url":null,"abstract":"","PeriodicalId":19084,"journal":{"name":"Nature biotechnology","volume":"42 12","pages":"1793-1793"},"PeriodicalIF":33.1,"publicationDate":"2024-12-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142809375","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
An update on Cuban biotech 古巴生物技术的最新进展
IF 33.1 1区 生物学
Nature biotechnology Pub Date : 2024-12-11 DOI: 10.1038/s41587-024-02458-y
José A. Buxadó, Miladys Limonta Fernández, Gerardo E. Guillén Nieto, Raimundo Ubieta Gómez, Marta Ayala Ávila
{"title":"An update on Cuban biotech","authors":"José A. Buxadó,&nbsp;Miladys Limonta Fernández,&nbsp;Gerardo E. Guillén Nieto,&nbsp;Raimundo Ubieta Gómez,&nbsp;Marta Ayala Ávila","doi":"10.1038/s41587-024-02458-y","DOIUrl":"10.1038/s41587-024-02458-y","url":null,"abstract":"","PeriodicalId":19084,"journal":{"name":"Nature biotechnology","volume":"42 12","pages":"1768-1770"},"PeriodicalIF":33.1,"publicationDate":"2024-12-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142809376","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Evo learns biological complexity from the molecular to genome scale 超能力从分子到基因组级别学习生物复杂性
IF 33.1 1区 生物学
Nature biotechnology Pub Date : 2024-12-11 DOI: 10.1038/s41587-024-02514-7
Iris Marchal
{"title":"Evo learns biological complexity from the molecular to genome scale","authors":"Iris Marchal","doi":"10.1038/s41587-024-02514-7","DOIUrl":"10.1038/s41587-024-02514-7","url":null,"abstract":"","PeriodicalId":19084,"journal":{"name":"Nature biotechnology","volume":"42 12","pages":"1793-1793"},"PeriodicalIF":33.1,"publicationDate":"2024-12-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142809371","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Plant bling: cellulose that sparkles 植物光泽:会发光的纤维素
IF 33.1 1区 生物学
Nature biotechnology Pub Date : 2024-12-11 DOI: 10.1038/s41587-024-02507-6
Michael Francisco
{"title":"Plant bling: cellulose that sparkles","authors":"Michael Francisco","doi":"10.1038/s41587-024-02507-6","DOIUrl":"10.1038/s41587-024-02507-6","url":null,"abstract":"","PeriodicalId":19084,"journal":{"name":"Nature biotechnology","volume":"42 12","pages":"1762-1762"},"PeriodicalIF":33.1,"publicationDate":"2024-12-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.com/articles/s41587-024-02507-6.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142809370","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Stem cells 干细胞
IF 33.1 1区 生物学
Nature biotechnology Pub Date : 2024-12-11 DOI: 10.1038/s41587-024-02496-6
{"title":"Stem cells","authors":"","doi":"10.1038/s41587-024-02496-6","DOIUrl":"10.1038/s41587-024-02496-6","url":null,"abstract":"Recent patents relating to compositions, methods of preparation and use of stem cells.","PeriodicalId":19084,"journal":{"name":"Nature biotechnology","volume":"42 12","pages":"1792-1792"},"PeriodicalIF":33.1,"publicationDate":"2024-12-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142809373","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
CRISPR unlocks a sweet tomato that keeps its size CRISPR解锁了保持其大小的甜番茄
IF 33.1 1区 生物学
Nature biotechnology Pub Date : 2024-12-11 DOI: 10.1038/s41587-024-02510-x
Iris Marchal
{"title":"CRISPR unlocks a sweet tomato that keeps its size","authors":"Iris Marchal","doi":"10.1038/s41587-024-02510-x","DOIUrl":"10.1038/s41587-024-02510-x","url":null,"abstract":"","PeriodicalId":19084,"journal":{"name":"Nature biotechnology","volume":"42 12","pages":"1793-1793"},"PeriodicalIF":33.1,"publicationDate":"2024-12-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142809372","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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