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本文引用的文献

1
Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B.用依特那考基因德扎帕罗韦克治疗B型血友病的基因疗法。
N Engl J Med. 2023 Feb 23;388(8):706-718. doi: 10.1056/NEJMoa2211644.
2
Subgenomic particles in rAAV vectors result from DNA lesion/break and non-homologous end joining of vector genomes.重组腺相关病毒（rAAV）载体中的亚基因组颗粒是由载体基因组的DNA损伤/断裂和非同源末端连接产生的。
Mol Ther Nucleic Acids. 2022 Aug 24;29:852-861. doi: 10.1016/j.omtn.2022.08.027. eCollection 2022 Sep 13.
3
Gene Therapy Advances: A Meta-Analysis of AAV Usage in Clinical Settings.基因治疗进展：临床环境中腺相关病毒使用情况的荟萃分析。
Front Med (Lausanne). 2022 Feb 9;8:809118. doi: 10.3389/fmed.2021.809118. eCollection 2021.
4
Long-Term Follow-Up of Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.脑肾上腺脑白质营养不良的造血干细胞基因治疗的长期随访。
Hum Gene Ther. 2021 Oct;32(19-20):1260-1269. doi: 10.1089/hum.2021.053. Epub 2021 May 4.
5
Liver Injury Increases the Incidence of HCC following AAV Gene Therapy in Mice.肝损伤增加了 AAV 基因治疗后小鼠 HCC 的发生率。
Mol Ther. 2021 Feb 3;29(2):680-690. doi: 10.1016/j.ymthe.2020.10.018. Epub 2020 Oct 22.
6
LncRNA MEG8 plays an oncogenic role in hepatocellular carcinoma progression through miR-367-3p/14-3-3ζ/TGFβR1 axis.长链非编码RNA MEG8通过miR-367-3p/14-3-3ζ/TGFβR1轴在肝细胞癌进展中发挥致癌作用。
Neoplasma. 2021 Mar;68(2):273-282. doi: 10.4149/neo_2020_200730N785. Epub 2020 Nov 5.
7
Gene Therapy for Hemophilia: Facts and Quandaries in the 21st Century.血友病的基因治疗：21世纪的事实与困境
Mediterr J Hematol Infect Dis. 2020 Sep 1;12(1):e2020069. doi: 10.4084/MJHID.2020.069. eCollection 2020.
8
Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B.首例体内血管内给药的腺相关病毒基因治疗的长期随访：用于严重血友病 B 的 AAV2-hFIX16。
Mol Ther. 2020 Sep 2;28(9):2073-2082. doi: 10.1016/j.ymthe.2020.06.001. Epub 2020 Jun 10.
9
Hepatocellular carcinoma in non-cirrhotic liver: A comprehensive review.非肝硬化肝脏中的肝细胞癌：全面综述
World J Hepatol. 2019 Jan 27;11(1):1-18. doi: 10.4254/wjh.v11.i1.1.
10
Incidence and risk factors for hepatocellular cancer in individuals with haemophilia: A National Inpatient Sample Study.血友病患者肝细胞癌的发病率和危险因素：国家住院患者样本研究。
Haemophilia. 2019 Mar;25(2):221-228. doi: 10.1111/hae.13668. Epub 2019 Jan 7.

Molecular evaluation and vector integration analysis of HCC complicating AAV gene therapy for hemophilia B.

作者信息

Schmidt Manfred, Foster Graham R, Coppens Michiel, Thomsen Hauke, Dolmetsch Ricardo, Heijink Liesbeth, Monahan Paul E, Pipe Steven W

机构信息

GeneWerk GmbH, Heidelberg, Germany.

Barts Liver Centre, Queen Mary University of London, London, United Kingdom.

出版信息

Blood Adv. 2023 Sep 12;7(17):4966-4969. doi: 10.1182/bloodadvances.2023009876.

DOI:10.1182/bloodadvances.2023009876
PMID:37352263
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10463188/
Abstract
摘要