Single Vector AAV Approach to Genome Editing in Pompe Disease

被引：0
|
作者：
Arnson, Benjamin [1 ]
Wang, Jason [2 ]
Courtney, David [3 ]
Han, Sang-oh [1 ]
Li, Songtao [1 ]
Cullen, Bryan R. [3 ]
Bursac, Nenad [2 ]
Koeberl, Dwight [1 ]
机构：
[1] Duke Univ, Pediat Med Genet, Durham, NC USA
[2] Duke Univ, Biomed Engn, Durham, NC USA
[3] Duke Univ, Mol Genet & Microbiol, Durham, NC USA
来源：
MOLECULAR THERAPY | 2021年 / 29卷 / 04期
关键词：
D O I：
暂无
中图分类号：
Q81 [生物工程学（生物技术）]; Q93 [微生物学];
学科分类号：
071005 ; 0836 ; 090102 ; 100705 ;
摘要：
600
引用
收藏
页码：291 / 291
页数：1
相关论文
共 50 条
  • [41] A removable virus vector suitable for plant genome editing
    Chujo, Tetsuya
    Yoshikawa, Manabu
    Ariga, Hirotaka
    Endo, Masaki
    Toki, Seiichi
    Ishibashi, Kazuhiro
    PLANT JOURNAL, 2017, 91 (03): : 558 - 561
  • [42] Current status of genome editing in vector mosquitoes: A review
    Reegan, Appadurai Daniel
    Ceasar, Stanislaus Antony
    Paulraj, Michael Gabriel
    Ignacimuthu, Savarimuthu
    Al-Dhabi, Naif Abdullah
    BioScience Trends, 2016, 10 (06) : 424 - 432
  • [43] Characterization of DNA Integrity for Recombinant AAV Vector with Oversize Genome
    Dong, Biao
    Lu, Hui
    Xiao, Weidong
    MOLECULAR THERAPY, 2009, 17 : S147 - S147
  • [44] In Vivo Genome Editing as a Therapeutic Approach
    Ho, Beatrice Xuan
    Loh, Sharon Jia Hui
    Chan, Woon Khiong
    Soh, Boon Seng
    INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 2018, 19 (09)
  • [45] Enabling redosing of AAV by immune management in Pompe disease: Preclinical to clinical studies
    Corti, Manuela
    Cleaver, Brian
    Clement, Nathalie
    Wang, Genscheng
    Benson, Janet
    Tarantal, Alice
    Herzog, Roland
    Byrne, Barry
    MOLECULAR GENETICS AND METABOLISM, 2018, 123 (02) : S34 - S34
  • [46] AAV Therapy Attenuates Respiratory Dysfunction in a Novel Rat Model of Pompe Disease
    Mahajan, Bhuvna
    Vaught, Lauren A.
    Todd, A. Gary
    Duncanson, Lauren A.
    Doyle, Brendan
    Fuller, David D.
    Byrne, Barry J.
    Falk, Darin J.
    MOLECULAR THERAPY, 2018, 26 (05) : 166 - 166
  • [47] Efforts to enhance AAV8-mediated gene therapy of Pompe disease
    Ziegler, R. J.
    Bercury, S. D.
    Zhu, Y.
    Foley, J. W.
    Gumlaw, N.
    Koeberl, D. D.
    Scheule, R. K.
    Cheng, S. H.
    CLINICAL THERAPEUTICS, 2008, 30 : S37 - S37
  • [48] Generation of infantile- and late-onset Pompe disease models using CRISPR-Cas9 genome editing
    Huang, Jeffrey Y.
    Wang, Raymond Y.
    MOLECULAR GENETICS AND METABOLISM, 2017, 120 (1-2) : S67 - S67
  • [49] Efforts to enhance AAV8-mediated gene therapy of Pompe disease
    Zieler, R.
    Bercury, S.
    Zhu, Y.
    Foley, J.
    Glimlaw, N.
    Koeberl, D.
    Scheule, R.
    Cheng, S.
    NEUROMUSCULAR DISORDERS, 2007, 17 (9-10) : 890 - 890
  • [50] A Highly Efficient Dual AAV Technology for Therapeutic (epi)Genome Editing Applications
    Riedmayr, Lisa M.
    Hinrichsmeyer, Klara S.
    Boehm, Sybille
    Splith, Victoria
    Michalakis, Stylianos
    Biel, Martin
    Becirovic, Elvir
    MOLECULAR THERAPY, 2020, 28 (04) : 13 - 13
12345