用于心脏、肺和血液疾病的胎猴基因转移中心：基因治疗界的 NHLBI 资源。
Center for fetal monkey gene transfer for heart, lung, and blood diseases: an NHLBI resource for the gene therapy community.
机构信息
Center for Fetal Monkey Gene Transfer for Heart, Lung, and Blood Diseases, University of California, Davis, 95616, USA.
出版信息
Hum Gene Ther. 2012 Nov;23(11):1130-5. doi: 10.1089/hum.2012.178. Epub 2012 Oct 19.
Abstract
The goals of the National Heart, Lung, and Blood Institute (NHLBI) Center for Fetal Monkey Gene Transfer for Heart, Lung, and Blood Diseases are to conduct gene transfer studies in monkeys to evaluate safety and efficiency; and to provide NHLBI-supported investigators with expertise, resources, and services to actively pursue gene transfer approaches in monkeys in their research programs. NHLBI-supported projects span investigators throughout the United States and have addressed novel approaches to gene delivery; "proof-of-principle"; assessed whether findings in small-animal models could be demonstrated in a primate species; or were conducted to enable new grant or IND submissions. The Center for Fetal Monkey Gene Transfer for Heart, Lung, and Blood Diseases successfully aids the gene therapy community in addressing regulatory barriers, and serves as an effective vehicle for advancing the field.
摘要
美国国立心肺血液研究所（NHLBI）胎儿猴基因转移中心的目标是在猴子身上进行基因转移研究，以评估安全性和效率；并为 NHLBI 支持的研究人员提供专业知识、资源和服务，以在他们的研究计划中积极采用猴子的基因转移方法。NHLBI 支持的项目涵盖了美国各地的研究人员，涉及基因传递的新方法；“原理验证”；评估小型动物模型中的发现是否可以在灵长类动物物种中得到证明；或进行新的资助或 IND 提交。胎儿猴基因转移中心为心脏、肺和血液疾病的基因治疗社区成功地解决了监管障碍，并成为推动该领域发展的有效手段。
相似文献
1
Center for fetal monkey gene transfer for heart, lung, and blood diseases: an NHLBI resource for the gene therapy community.用于心脏、肺和血液疾病的胎猴基因转移中心：基因治疗界的 NHLBI 资源。
Hum Gene Ther. 2012 Nov;23(11):1130-5. doi: 10.1089/hum.2012.178. Epub 2012 Oct 19.
2
The Burden of Heart, Lung, and Blood Diseases in the United States, 1990 to 2016: Perspectives from the National Heart, Lung, and Blood Institute.1990年至2016年美国心脏、肺和血液疾病负担：来自国家心脏、肺和血液研究所的观点。
Glob Heart. 2017 Dec;12(4):349-358. doi: 10.1016/j.gheart.2017.11.001.
3
Critical resources for gene therapy in Heart, Lung, and Blood Diseases Working Group.心脏、肺和血液疾病工作组基因治疗的关键资源。
Mol Ther. 2006 Apr;13(4):641-3.
4
National Heart, Lung, and Blood Institute resources and programs for gene therapy in heart, lung, and blood diseases.美国国立心肺血液研究所关于心脏、肺部和血液疾病基因治疗的资源与项目。
Circ Res. 2007 Jun 22;100(12):1667-9. doi: 10.1161/CIRCRESAHA.107.101201.
5
NHLBI supports diverse research.美国国立心肺血液研究所支持多样化的研究。
US Med. 1979 Jan 15;15(2):57-8.
6
Cell therapy for lung diseases. Report from an NIH-NHLBI workshop, November 13-14, 2012.细胞疗法治疗肺部疾病。NIH-NHLBI 研讨会报告，2012 年 11 月 13 日至 14 日。
Am J Respir Crit Care Med. 2013 Aug 1;188(3):370-5. doi: 10.1164/rccm.201303-0522WS.
7
The National Heart, Lung, and Blood Institute-funded Production Assistance for Cellular Therapies (PACT) program: Eighteen years of cell therapy.美国国立心肺血液研究所资助的细胞治疗生产援助 (PACT) 计划：18 年的细胞治疗。
Clin Transl Sci. 2021 Nov;14(6):2099-2110. doi: 10.1111/cts.13102. Epub 2021 Jul 19.
8
Molecular genetics of heart, lung, and blood diseases. The shape of things to come.心脏、肺和血液疾病的分子遗传学。未来的发展趋势。
Circulation. 1993 Dec;88(6):2487-8. doi: 10.1161/01.cir.88.6.2487.
9
National Heart, Lung, and Blood Institute Workshop Summary: Enhancing Opportunities for Training and Retention of a Diverse Biomedical Workforce.美国国立心肺血液研究所研讨会总结：增加培训机会并留住多元化生物医学人才队伍。
Ann Am Thorac Soc. 2016 Apr;13(4):562-7. doi: 10.1513/AnnalsATS.201509-624OT.
10
The National Heart, Lung, and Blood Institute Strategic Vision Implementation for Health Equity Research.美国国家心肺血液研究所健康公平研究战略愿景的实施。
Ethn Dis. 2019 Feb 21;29(Suppl 1):57-64. doi: 10.18865/ed.29.S1.57. eCollection 2019.
引用本文的文献
1
Nonhuman Primates in Translational Research.非人类灵长类动物在转化研究中的应用。
Annu Rev Anim Biosci. 2022 Feb 15;10:441-468. doi: 10.1146/annurev-animal-021419-083813.
2
Dosing and Re-Administration of Lentiviral Vector for Gene Therapy in Rhesus Monkeys and ADA-Deficient Mice.恒河猴和腺苷脱氨酶缺乏症小鼠基因治疗中慢病毒载体的给药与重新给药
Mol Ther Methods Clin Dev. 2019 Nov 16;16:78-93. doi: 10.1016/j.omtm.2019.11.004. eCollection 2020 Mar 13.
3
Safe and Sustained Expression of Human Iduronidase After Intrathecal Administration of Adeno-Associated Virus Serotype 9 in Infant Rhesus Monkeys.鞘内注射腺相关病毒血清型 9 后人类艾杜糖苷酸酶在婴儿恒河猴中的安全和持续表达。
Hum Gene Ther. 2019 Aug;30(8):957-966. doi: 10.1089/hum.2019.012. Epub 2019 Jun 10.
4
Microglia: An Intrinsic Component of the Proliferative Zones in the Fetal Rhesus Monkey (Macaca mulatta) Cerebral Cortex.小胶质细胞：恒河猴（Macaca mulatta）胎儿大脑皮质增殖区的固有组成部分。
Cereb Cortex. 2019 Jul 5;29(7):2782-2796. doi: 10.1093/cercor/bhy145.
5
Systemic and Persistent Muscle Gene Expression in Rhesus Monkeys with a Liver De-Targeted Adeno-Associated Virus Vector.使用肝脏脱靶腺相关病毒载体的恒河猴全身持续性肌肉基因表达
Hum Gene Ther. 2017 May;28(5):385-391. doi: 10.1089/hum.2016.130. Epub 2017 Jan 25.
6
Transfer of Therapeutic Genes into Fetal Rhesus Monkeys Using Recombinant Adeno-Associated Type I Viral Vectors.使用重组I型腺相关病毒载体将治疗性基因导入恒河猴胎儿
Hum Gene Ther Clin Dev. 2016 Dec;27(4):152-159. doi: 10.1089/humc.2016.119.
7
Development of operational immunologic tolerance with neonatal gene transfer in nonhuman primates: preliminary studies.非人灵长类动物新生期基因转移诱导可操作性免疫耐受的研究：初步研究
Gene Ther. 2015 Nov;22(11):923-30. doi: 10.1038/gt.2015.65. Epub 2015 Aug 23.
8
Effects of vector backbone and pseudotype on lentiviral vector-mediated gene transfer: studies in infant ADA-deficient mice and rhesus monkeys.载体骨架和假型对慢病毒载体介导的基因转移的影响：在婴儿腺苷脱氨酶缺乏症小鼠和恒河猴中的研究。
Mol Ther. 2014 Oct;22(10):1803-16. doi: 10.1038/mt.2014.88. Epub 2014 Jun 13.
9
Myeloid-lymphoid ontogeny in the rhesus monkey (Macaca mulatta).恒河猴（猕猴）的髓系-淋巴系个体发生
Anat Rec (Hoboken). 2014 Aug;297(8):1392-406. doi: 10.1002/ar.22943. Epub 2014 May 28.
10
Adeno-associated virus-mediated gene therapy for metabolic myopathy.腺相关病毒介导的代谢性肌病基因治疗。
Hum Gene Ther. 2013 Nov;24(11):928-36. doi: 10.1089/hum.2013.2514.
本文引用的文献
1
Radiolabeling human peripheral blood stem cells for positron emission tomography (PET) imaging in young rhesus monkeys.放射性标记人外周血干细胞进行正电子发射断层扫描（PET）成像在年轻恒河猴。
PLoS One. 2013 Oct 3;8(10):e77148. doi: 10.1371/journal.pone.0077148. eCollection 2013.
2
The gene therapy journey for hemophilia: are we there yet?基因治疗血友病之旅：我们到了吗？
Blood. 2012 Nov 29;120(23):4482-7. doi: 10.1182/blood-2012-05-423210. Epub 2012 Jul 24.
3
Challenges in vector and trial design using retroviral vectors for long-term gene correction in hematopoietic stem cell gene therapy.在造血干细胞基因治疗中使用逆转录病毒载体进行长期基因校正时，载体和试验设计面临的挑战。
Mol Ther. 2012 Jun;20(6):1084-94. doi: 10.1038/mt.2012.93.
4
Decade-long safety and function of retroviral-modified chimeric antigen receptor T cells.逆转录病毒修饰的嵌合抗原受体 T 细胞的十年安全性和功能。
Sci Transl Med. 2012 May 2;4(132):132ra53. doi: 10.1126/scitranslmed.3003761.
5
AAV2 gene therapy readministration in three adults with congenital blindness.三种成人先天性失明患者接受 AAV2 基因治疗再给药。
Sci Transl Med. 2012 Feb 8;4(120):120ra15. doi: 10.1126/scitranslmed.3002865.
6
Nonmyeloablative conditioning regimen to increase engraftment of gene-modified hematopoietic stem cells in young rhesus monkeys.非清髓预处理方案增加基因修饰的造血干细胞在幼年恒河猴中的植入。
Mol Ther. 2012 May;20(5):1033-45. doi: 10.1038/mt.2011.312. Epub 2012 Jan 31.
7
Changes in the frequency and in vivo vessel-forming ability of rhesus monkey circulating endothelial colony-forming cells across the lifespan (birth to aged).恒河猴循环内皮祖细胞在整个生命周期（出生到老年）中的频率和体内血管生成能力的变化。
Pediatr Res. 2012 Feb;71(2):156-61. doi: 10.1038/pr.2011.22. Epub 2011 Dec 21.
8
AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage.在围生期进行 AAV 介导的基因转移可导致 FVII 表达水平升高，从而预防致命性自发性出血。
Blood. 2012 Jan 26;119(4):957-66. doi: 10.1182/blood-2011-09-377630. Epub 2011 Dec 1.
9
Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years.针对由RPE65基因突变引起的莱伯先天性黑蒙的基因治疗：15名儿童和成人的安全性和有效性，随访长达3年。
Arch Ophthalmol. 2012 Jan;130(1):9-24. doi: 10.1001/archophthalmol.2011.298. Epub 2011 Sep 12.
10
AAV8-mediated hepatic gene transfer in infant rhesus monkeys (Macaca mulatta).腺相关病毒 8 介导的婴儿恒河猴（Macaca mulatta）肝基因转移。
Mol Ther. 2011 Nov;19(11):2012-20. doi: 10.1038/mt.2011.151. Epub 2011 Aug 2.
Zotero 插件