研究报告 |
|
|
|
|
AAV载体介导的蓬佩病模型小鼠体内基因治疗研究* |
武志杰1,2,马文豪2,董哲岳2,吴小兵2,**(),杨怡姝1,**(),盛望1 |
1. 北京工业大学 北京 100124 2. 北京锦篮基因科技有限公司 北京 100176 |
|
AAV Vector Mediated Gene Therapy in Pompe Model Mice |
Zhi-jie WU1,2,Wen-hao MA2,Zhe-yue DONG2,Xiao-bing WU2,**(),Yi-shu YANG1,**(),Wang SHENG1 |
1. Beijing University of Technology, Beijing 100124,China 2. Beijing GeneCradle Pharmaceutical Co Ltd, Beijing 100176,China |
引用本文:
武志杰,马文豪,董哲岳,吴小兵,杨怡姝,盛望. AAV载体介导的蓬佩病模型小鼠体内基因治疗研究*[J]. 中国生物工程杂志, 2022, 42(7): 24-34.
Zhi-jie WU,Wen-hao MA,Zhe-yue DONG,Xiao-bing WU,Yi-shu YANG,Wang SHENG. AAV Vector Mediated Gene Therapy in Pompe Model Mice. China Biotechnology, 2022, 42(7): 24-34.
链接本文:
https://manu60.magtech.com.cn/biotech/CN/10.13523/j.cb.2202040
或
https://manu60.magtech.com.cn/biotech/CN/Y2022/V42/I7/24
|
[1] |
Kohler L, Puertollano R, Raben N. Pompe disease: from basic science to therapy. Neurotherapeutics, 2018, 15(4): 928-942.
doi: 10.1007/s13311-018-0655-y
pmid: 30117059
|
[2] |
Taverna S, Cammarata G, Colomba P, et al. Pompe disease: pathogenesis, molecular genetics and diagnosis. Aging, 2020, 12(15): 15856-15874.
doi: 10.18632/aging.103794
|
[3] |
中华医学会儿科学分会内分泌遗传代谢学组, 中华医学会医学遗传学分会, 中华医学会儿科学分会罕见病学组, 等. 儿童糖原累积病Ⅱ型诊断及治疗中国专家共识. 中华儿科杂志, 2021, 59(6): 439-445.
|
|
The Subspecialty Group of Endocrinologic, Hereditary and Metabolic Diseases, the Society of Pediatrics, Chinese Medical Association; the Society of Medical Genetics, Chinese Medical Association; the Subspecialty Group of Rare Diseases, the Society of Pediatrics, Chinese Medical Association, et al. Chinese experts consensus on diagnosis and treatment of glycogen storage disease type Ⅱ in children. Chinese Journal of Pediatrics, 2021, 59(6): 439-445.
|
[4] |
Colella P, Mingozzi F. Gene therapy for pompe disease: the time is now. Human Gene Therapy, 2019, 30(10): 1245-1262.
doi: 10.1089/hum.2019.109
|
[5] |
Kishnani P S, Hwu W L, Mandel H, et al. A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease. The Journal of Pediatrics, 2006, 148(5): 671-676.e2.
doi: 10.1016/j.jpeds.2005.11.033
|
[6] |
van Capelle C I, van der Meijden J C, van den Hout J M P, et al. Childhood Pompe disease: clinical spectrum and genotype in 31 patients. Orphanet Journal of Rare Diseases, 2016, 11(1): 65.
doi: 10.1186/s13023-016-0442-y
pmid: 27189384
|
[7] |
Preisler N, Lukacs Z, Vinge L, et al. Late-onset Pompe disease is prevalent in unclassified limb-girdle muscular dystrophies. Molecular Genetics and Metabolism, 2013, 110(3): 287-289.
doi: 10.1016/j.ymgme.2013.08.005
pmid: 24011652
|
[8] |
Falk D J, Soustek M S, Todd A G, et al. Comparative impact of AAV and enzyme replacement therapy on respiratory and cardiac function in adult Pompe mice. Molecular Therapy - Methods & Clinical Development, 2015, 2: 15007.
|
[9] |
Costa-Verdera H, Collaud F, Riling C R, et al. Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human Primates. Nature Communications, 2021, 12: 6393.
doi: 10.1038/s41467-021-26744-4
pmid: 34737297
|
[10] |
Zincarelli C, Soltys S, Rengo G, et al. Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection. Molecular Therapy, 2008, 16(6): 1073-1080.
doi: 10.1038/mt.2008.76
pmid: 18414476
|
[11] |
Yu Z, Zhou S, Luo N, et al. Three-phase partitioning combined with density gradient ultracentrifugation as an economic and universal process for large scale purification of AAV vectors. Molecular Therapy - Methods & Clinical Development, 2019, 17:34-48.
|
[12] |
Werling N J, Satkunanathan S, Thorpe R, et al. Systematic comparison and validation of quantitative real-time PCR methods for the quantitation of adeno-associated viral products. Human Gene Therapy Methods, 2015, 26(3): 82-92.
doi: 10.1089/hgtb.2015.013
|
[13] |
Raben N, Nagaraju K, Lee E, et al. Targeted disruption of the acid alpha-glucosidase gene in mice causes an illness with critical features of both infantile and adult human glycogen storage disease type II. The Journal of Biological Chemistry, 1998, 273(30): 19086-19092.
doi: 10.1074/jbc.273.30.19086
|
[14] |
马文豪, 章嫣, 董哲岳, 等. 携带CAR启动子的重组AAV9病毒在小鼠体内表达分布特性研究. 病毒学报, 2019, 35(3): 423-430.
|
|
Ma W H, Zhang Y, Dong Z Y, et al. Biodistribution of adeno-associated virus 9-mediated gene expression with a CAR promoter in mice. Chinese Journal of Virology, 2019, 35(3): 423-430.
|
[15] |
Kishnani P S, Nicolino M, Voit T, et al. Chinese hamster ovary cell-derived recombinant human acid α-glucosidase in infantile-onset Pompe disease. The Journal of Pediatrics, 2006, 149(1): 89-97.
doi: 10.1016/j.jpeds.2006.02.035
|
[16] |
Chan J, Desai A K, Kazi Z B, et al. The emerging phenotype of late-onset Pompe disease: a systematic literature review. Molecular Genetics and Metabolism, 2017, 120(3): 163-172.
doi: 10.1016/j.ymgme.2016.12.004
|
[17] |
Samulski R J, Muzyczka N. AAV-mediated gene therapy for research and therapeutic purposes. Annual Review of Virology, 2014, 1(1): 427-451.
doi: 10.1146/annurev-virology-031413-085355
pmid: 26958729
|
[18] |
Verdera H C, Kuranda K, Mingozzi F. AAV vector immunogenicity in humans: a long journey to successful gene transfer. Molecular Therapy, 2020, 28(3): 723-746.
doi: S1525-0016(20)30003-4
pmid: 31972133
|
[19] |
Weber T. Anti-AAV antibodies in AAV gene therapy: current challenges and possible solutions. Frontiers in Immunology, 2021, 12: 658399.
doi: 10.3389/fimmu.2021.658399
|
[20] |
Salabarria S M, Nair J, Clement N, et al. Advancements in AAV-mediated gene therapy for pompe disease. Journal of Neuromuscular Diseases, 2020, 7(1): 15-31.
doi: 10.3233/JND-190426
pmid: 31796685
|
[21] |
ElMallah M K, Falk D J, Nayak S, et al. Sustained correction of motoneuron histopathology following intramuscular delivery of AAV in pompe mice. Molecular Therapy, 2014, 22(4): 702-712.
doi: 10.1038/mt.2013.282
|
[22] |
Falk D J, Mah C S, Soustek M S, et al. Intrapleural administration of AAV9 improves neural and cardiorespiratory function in Pompe disease. Molecular Therapy, 2013, 21(9): 1661-1667.
doi: 10.1038/mt.2013.96
|
[23] |
Amalfitano A, Mcvie-Wylie A J, Hu H, et al. Systemic correction of the muscle disorder glycogen storage disease type II after hepatic targeting of a modified adenovirus vector encoding human acid-alpha-glucosidase. Proceedings of the National Academy of Sciences of the United States of America, 1999, 96(16): 8861-8866.
|
[24] |
Han S O, Ronzitti G, Arnson B, et al. Low-dose liver-targeted gene therapy for pompe disease enhances therapeutic efficacy of ERT via immune tolerance induction. Molecular Therapy - Methods & Clinical Development, 2017, 4: 126-136.
|
[25] |
Ronzitti G, Collaud F, Laforet P, et al. Progress and challenges of gene therapy for Pompe disease. Annals of Translational Medicine, 2019, 7(13): 287.
doi: 10.21037/atm.2019.04.67
|
|
Viewed |
|
|
|
Full text
|
|
|
|
|
Abstract
|
|
|
|
|
Cited |
|
|
|
|
|
Shared |
|
|
|
|
|
Discussed |
|
|
|
|