A Highly Efficient Dual AAV Technology for Therapeutic (epi)Genome Editing Applications

被引:0
|
作者
Riedmayr, Lisa M. [1 ,2 ]
Hinrichsmeyer, Klara S. [1 ,2 ]
Boehm, Sybille [1 ,2 ]
Splith, Victoria [1 ,2 ]
Michalakis, Stylianos [3 ]
Biel, Martin [1 ,2 ]
Becirovic, Elvir [1 ,2 ]
机构
[1] Ludwig Maximilians Univ Munchen, Pharm, Munich, Germany
[2] Ludwig Maximilians Univ Munchen, Ctr Integrated Prot Sci Munich CiPSM, Munich, Germany
[3] Ludwig Maximilians Univ Munchen, Univ Eye Hosp, Munich, Germany
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
25
引用
收藏
页码:13 / 13
页数:1
相关论文
共 50 条
  • [1] mRNA trans-splicing dual AAV vectors for (epi)genome editing and gene therapy
    Lisa Maria Riedmayr
    Klara Sonnie Hinrichsmeyer
    Stefan Bernhard Thalhammer
    David Manuel Mittas
    Nina Karguth
    Dina Yehia Otify
    Sybille Böhm
    Valentin Johannes Weber
    Michael David Bartoschek
    Victoria Splith
    Manuela Brümmer
    Raphael Ferreira
    Nanda Boon
    Gabriele Maria Wögenstein
    Christian Grimm
    Jan Wijnholds
    Verena Mehlfeld
    Stylianos Michalakis
    Stefanie Fenske
    Martin Biel
    Elvir Becirovic
    [J]. Nature Communications, 14
  • [2] mRNA trans-splicing dual AAV vectors for (epi)genome editing and gene therapy
    Riedmayr, Lisa Maria
    Hinrichsmeyer, Klara Sonnie
    Thalhammer, Stefan Bernhard
    Mittas, David Manuel
    Karguth, Nina
    Otify, Dina Yehia
    Boehm, Sybille
    Weber, Valentin Johannes
    Bartoschek, Michael David
    Splith, Victoria
    Bruemmer, Manuela
    Ferreira, Raphael
    Boon, Nanda
    Woegenstein, Gabriele Maria
    Grimm, Christian
    Wijnholds, Jan
    Mehlfeld, Verena
    Michalakis, Stylianos
    Fenske, Stefanie
    Biel, Martin
    Becirovic, Elvir
    [J]. NATURE COMMUNICATIONS, 2023, 14 (01)
  • [3] Efficient therapeutic genome editing in the horse
    Pinzon, C.
    Snyder, M.
    Lazzarotto, C.
    Skenandore, C.
    Hernandez, C.
    Moreno, N.
    Horn, D.
    Golding, M.
    Varner, D. D.
    Long, C.
    [J]. TRANSGENIC RESEARCH, 2018, 27 (05) : 482 - 482
  • [4] Genome Editing Techniques and Their Therapeutic Applications
    Calos, M. P.
    [J]. CLINICAL PHARMACOLOGY & THERAPEUTICS, 2017, 101 (01) : 42 - 51
  • [5] Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editing
    Trevor J. Gonzalez
    Katherine E. Simon
    Leo O. Blondel
    Marco M. Fanous
    Angela L. Roger
    Maribel Santiago Maysonet
    Garth W. Devlin
    Timothy J. Smith
    Daniel K. Oh
    L. Patrick Havlik
    Ruth M. Castellanos Rivera
    Jorge A. Piedrahita
    Mai K. ElMallah
    Charles A. Gersbach
    Aravind Asokan
    [J]. Nature Communications, 13
  • [6] Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editing
    Gonzalez, Trevor J.
    Simon, Katherine E.
    Blondel, Leo O.
    Fanous, Marco M.
    Roger, Angela L.
    Maysonet, Maribel Santiago
    Devlin, Garth W.
    Smith, Timothy J.
    Oh, Daniel K.
    Havlik, L. Patrick
    Castellanos Rivera, Ruth M.
    Piedrahita, Jorge A.
    ElMallah, Mai K.
    Gersbach, Charles A.
    Asokan, Aravind
    [J]. NATURE COMMUNICATIONS, 2022, 13 (01)
  • [7] Genome editing technology and medical applications
    Liren Wang
    Bin Zhou
    Dali Li
    [J]. Science China(Life Sciences), 2024, 67 (12) : 2537 - 2539
  • [8] Emerging therapeutic applications of CRISPR genome editing
    Qasim, Waseem
    [J]. EMERGING TOPICS IN LIFE SCIENCES, 2019, 3 (03) : 257 - 260
  • [9] Synthetic sgRNA enables highly efficient CRISPR editing of primary cells for therapeutic applications
    Holden, K.
    Maures, T.
    Walker, J.
    Bak, R.
    House, I.
    Wiebking, V.
    [J]. HUMAN GENE THERAPY, 2017, 28 (12) : A66 - A66
  • [10] Highly Efficient Genome Editing for Crop Improvement.
    Clasen, Benjamin
    [J]. IN VITRO CELLULAR & DEVELOPMENTAL BIOLOGY-PLANT, 2015, 51 (04) : 492 - 492