我通往腺相关病毒及腺相关病毒基因治疗之路：个人视角 - Suppr

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My Pathway to Adeno-Associated Virus and Adeno-Associated Virus Gene Therapy: A Personal Perspective.

作者信息

Carter Barrie J

机构信息

Seattle, Washington, USA.

出版信息

Hum Gene Ther. 2020 May;31(9-10):494-498. doi: 10.1089/hum.2020.29120.bca. Epub 2020 Apr 9.

DOI:10.1089/hum.2020.29120.bca

PMID:32275185

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC7238672/

Abstract

摘要

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a9ff/7238672/d11010111e2a/hum.2020.29120.bca_figure1.jpg

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Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A.AAV5-hFVIII-SQ 基因治疗血友病 A 的多年随访。

N Engl J Med. 2020 Jan 2;382(1):29-40. doi: 10.1056/NEJMoa1908490.

Gene Therapy with BMN 270 Results in Therapeutic Levels of FVIII in Mice and Primates and Normalization of Bleeding in Hemophilic Mice.BMN 270 基因治疗可在小鼠和灵长类动物中产生治疗水平的 FVIII，并使血友病小鼠出血正常化。

Mol Ther. 2018 Feb 7;26(2):496-509. doi: 10.1016/j.ymthe.2017.12.009. Epub 2017 Dec 14.

AAV5-Factor VIII Gene Transfer in Severe Hemophilia A.AAV5-Factor VIII 基因治疗重度血友病 A。

N Engl J Med. 2017 Dec 28;377(26):2519-2530. doi: 10.1056/NEJMoa1708483. Epub 2017 Dec 9.

Development of rAAV2-CFTR: History of the First rAAV Vector Product to be Used in Humans.重组腺相关病毒2型-囊性纤维化跨膜传导调节因子（rAAV2-CFTR）的研发：首个用于人体的重组腺相关病毒载体产品的历程

Hum Gene Ther Methods. 2016 Apr;27(2):49-58. doi: 10.1089/hgtb.2015.150. Epub 2016 Feb 19.

Effect of gene therapy on visual function in Leber's congenital amaurosis.基因治疗对莱伯先天性黑蒙视觉功能的影响。

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Mol Ther. 2004 Dec;10(6):981-9. doi: 10.1016/j.ymthe.2004.09.011.

Genetic fate of recombinant adeno-associated virus vector genomes in muscle.重组腺相关病毒载体基因组在肌肉中的遗传命运。

J Virol. 2003 Mar;77(6):3495-504. doi: 10.1128/jvi.77.6.3495-3504.2003.

In vivo model of adeno-associated virus vector persistence and rescue.腺相关病毒载体持久性与拯救的体内模型

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J Virol. 1983 Feb;45(2):555-64. doi: 10.1128/JVI.45.2.555-564.1983.

Nucleotide sequence of the inverted terminal repetition in adeno-associated virus DNA.腺相关病毒DNA中反向末端重复序列的核苷酸序列。

J Virol. 1980 May;34(2):402-9. doi: 10.1128/JVI.34.2.402-409.1980.

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