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Hematopoietic Stem Cell-Targeted Gene Therapy Using Lentiviral Vectors 慢病毒载体的造血干细胞靶向基因治疗
Journal of Tokyo Medical University Pub Date : 2023-08-31 DOI: 10.1272/manms.19.205
Naoya Uchida
{"title":"Hematopoietic Stem Cell-Targeted Gene Therapy Using Lentiviral Vectors","authors":"Naoya Uchida","doi":"10.1272/manms.19.205","DOIUrl":"https://doi.org/10.1272/manms.19.205","url":null,"abstract":"Gene therapy targeting hematopoietic stem cells (HSCs) is a promising treatment for a variety of genetic disorders, including immunodeficiency, hemoglobinopathies, congenital cytopenia, and metabolic diseases. HSCs can reconstitute peripheral blood throughout life due to their capacity for self-renewal and their hematopoietic multipotency. This makes it possible to cure genetic diseases for an entire lifetime by replacing or repairing pathogenic mutations/deletions in HSCs. Autologous HSC-targeted gene therapies entailing lentiviral gene addition as well as gene editing are currently under development. These can be widely applied to most patients, as there is no requirement for a suitable donor. Current gene addition/editing therapies are based on harvesting the patient's CD34+ HSCs, performing gene modification ex vivo, and then transplanting the modified HSCs back into the patient. The efficacy of ex vivo lentiviral HSC gene therapy has been proved in recent trials; however, the ex vivo process requires a GMP-level cell processing center and is expensive, which limits its global application. It is therefore crucial to develop in vivo HSC gene therapies, in which a therapeutic gene or gene editing tools can be delivered directly into bone marrow HSCs via systemic administration without ex vivo culture. This manuscript presents an overview of the current HSC-targeted gene therapies using lentiviral vectors.","PeriodicalId":35560,"journal":{"name":"Journal of Tokyo Medical University","volume":"104 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-08-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135989976","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
泌尿器AI研究の現状 泌尿AI研究现状
Journal of Tokyo Medical University Pub Date : 2023-08-31 DOI: 10.1272/manms.19.283
Jun Akatsuka, Yukihiro Kondo
{"title":"泌尿器AI研究の現状","authors":"Jun Akatsuka, Yukihiro Kondo","doi":"10.1272/manms.19.283","DOIUrl":"https://doi.org/10.1272/manms.19.283","url":null,"abstract":"","PeriodicalId":35560,"journal":{"name":"Journal of Tokyo Medical University","volume":"10 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-08-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135989961","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Genome Integration of Adeno-associated Virus (AAV) Vectors into Infected Cells 腺相关病毒(AAV)载体在感染细胞中的基因组整合
Journal of Tokyo Medical University Pub Date : 2023-08-31 DOI: 10.1272/manms.19.211
Yukihiko Hirai
{"title":"Genome Integration of Adeno-associated Virus (AAV) Vectors into Infected Cells","authors":"Yukihiko Hirai","doi":"10.1272/manms.19.211","DOIUrl":"https://doi.org/10.1272/manms.19.211","url":null,"abstract":"The development of gene therapy products using adeno-associated virus (AAV) vectors is progressing, and the gene therapy market is rapidly expanding. AAV shows no pathogenicity in the human body, has extremely low cytotoxicity and, unlike lentiviral and retroviral vectors, rarely integrates into chromosomes. Consequently, risk associated with AAV was thought to be low. In recent years, however, adverse events such as hepatotoxicity have become apparent as cases accumulate among laboratory animals and in clinical trials. In this paper, we present an overview of the integration of AAV vectors into the genome of infected cells, which is thought to be the cause of adverse events.","PeriodicalId":35560,"journal":{"name":"Journal of Tokyo Medical University","volume":"31 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-08-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135989966","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Expectations for AI in Diagnostic Imaging 人工智能在诊断成像中的应用前景
Journal of Tokyo Medical University Pub Date : 2023-08-31 DOI: 10.1272/manms.19.286
Tadashi Machida
{"title":"Expectations for AI in Diagnostic Imaging","authors":"Tadashi Machida","doi":"10.1272/manms.19.286","DOIUrl":"https://doi.org/10.1272/manms.19.286","url":null,"abstract":"","PeriodicalId":35560,"journal":{"name":"Journal of Tokyo Medical University","volume":"16 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-08-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135989970","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
腎血栓性微小血管症の病理像を認めステロイドが奏功したTAFRO症候群の1例 肾血栓性微血管症的病理，类固醇奏效的TAFRO症候群的一个例子
Journal of Tokyo Medical University Pub Date : 2023-08-31 DOI: 10.1272/manms.19.252
Daisuke Miyamoto, Yukinao Sakai, Masato Iwabu
{"title":"腎血栓性微小血管症の病理像を認めステロイドが奏功したTAFRO症候群の1例","authors":"Daisuke Miyamoto, Yukinao Sakai, Masato Iwabu","doi":"10.1272/manms.19.252","DOIUrl":"https://doi.org/10.1272/manms.19.252","url":null,"abstract":"","PeriodicalId":35560,"journal":{"name":"Journal of Tokyo Medical University","volume":"4 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-08-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135989972","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Journal of Nippon Medical School Vol. 88, No. 5, No. 6,Vol. 89, No. 1 Summary 《日本医学院学报》第88卷第5期、第6期。89、1 .总结
Journal of Tokyo Medical University Pub Date : 2023-08-31 DOI: 10.1272/manms.19.270
{"title":"Journal of Nippon Medical School Vol. 88, No. 5, No. 6,Vol. 89, No. 1 Summary","authors":"","doi":"10.1272/manms.19.270","DOIUrl":"https://doi.org/10.1272/manms.19.270","url":null,"abstract":"","PeriodicalId":35560,"journal":{"name":"Journal of Tokyo Medical University","volume":"48 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-08-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135989973","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
日本医科大学におけるAI教育とAIを活用した生物物理研究 日本医科大学的AI教育和利用AI的生物物理研究
Journal of Tokyo Medical University Pub Date : 2023-08-31 DOI: 10.1272/manms.19.279
Hiroshi Fujisaki
{"title":"日本医科大学におけるAI教育とAIを活用した生物物理研究","authors":"Hiroshi Fujisaki","doi":"10.1272/manms.19.279","DOIUrl":"https://doi.org/10.1272/manms.19.279","url":null,"abstract":"","PeriodicalId":35560,"journal":{"name":"Journal of Tokyo Medical University","volume":"9 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-08-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135989965","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
History of Gene Therapy 基因治疗的历史
Journal of Tokyo Medical University Pub Date : 2023-08-31 DOI: 10.1272/manms.19.199
Takashi Shimada
{"title":"History of Gene Therapy","authors":"Takashi Shimada","doi":"10.1272/manms.19.199","DOIUrl":"https://doi.org/10.1272/manms.19.199","url":null,"abstract":"Once a dream cure for genetic diseases, gene therapy came to be considered potentially realizable with the advent of recombinant DNA technology in the 1970s. In the 1980s, viral vectors were developed, and ethical issues regarding human gene manipulation began to be addressed. In 1990, the world’s first gene therapy was launched in the United States. Since then, various gene therapies have been tried around the world, but have failed to show clear efficacy. In addition, safety problems, including a fatal accident and the development of leukemia, have occurred with gene therapy. Consequently, the once high expectations for gene therapy declined. Since the end of the 2000s, however, the efficacy of gene therapies for treatment of genetic diseases and cancer has been confirmed time and again, and expectations for gene therapy have been revived. At present, more than 20 gene therapy products have been approved, and gene therapies are now poised to become practical medicine.","PeriodicalId":35560,"journal":{"name":"Journal of Tokyo Medical University","volume":"32 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-08-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135989977","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Publication of "Evidence-based Guidebook of Routine Esophagogastroduodenoscopy" for Standardization of Upper Gastrointestinal Endoscopy 出版《食管胃十二指肠镜常规循证指南》，规范上消化道内镜检查
Journal of Tokyo Medical University Pub Date : 2023-04-20 DOI: 10.1272/manms.19.178
O. Goto, T. Akimoto, Naohiko Akimoto, J. Omori, Takeshi Onda, Kumiko Kirita, Eriko Koizumi, Hiroto Noda, Kazutoshi Higuchi, K. Iwakiri
{"title":"Publication of \"Evidence-based Guidebook of Routine Esophagogastroduodenoscopy\" for Standardization of Upper Gastrointestinal Endoscopy","authors":"O. Goto, T. Akimoto, Naohiko Akimoto, J. Omori, Takeshi Onda, Kumiko Kirita, Eriko Koizumi, Hiroto Noda, Kazutoshi Higuchi, K. Iwakiri","doi":"10.1272/manms.19.178","DOIUrl":"https://doi.org/10.1272/manms.19.178","url":null,"abstract":"","PeriodicalId":35560,"journal":{"name":"Journal of Tokyo Medical University","volume":"72 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-04-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"81580802","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
周産期医療と共に歩んだ40年 与围产期医疗同行的40年
Journal of Tokyo Medical University Pub Date : 2023-04-20 DOI: 10.1272/manms.19.121
章人 中井
{"title":"周産期医療と共に歩んだ40年","authors":"章人 中井","doi":"10.1272/manms.19.121","DOIUrl":"https://doi.org/10.1272/manms.19.121","url":null,"abstract":"","PeriodicalId":35560,"journal":{"name":"Journal of Tokyo Medical University","volume":"80 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-04-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"86244788","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","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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