AAV-Mediated Delivery of Single Base Editors Using Intein Trans-Splicing

被引：0
|
作者：
Winter, Jackson S. [1 ]
Luu, Alan [2 ,3 ]
Zeballos, Alejandra [1 ]
Gapinske, Michael [1 ]
Woods, Wendy S. [1 ]
Gaj, Thomas [1 ]
Song, Jun S. [2 ,3 ]
Perez-Pinera, Pablo [1 ,3 ,4 ,5 ]
机构：
[1] Univ Illinois, Bioengn, Urbana, IL USA
[2] Univ Illinois, Phys, Urbana, IL USA
[3] Carl R Woese Inst Genom Biol, Urbana, IL USA
[4] Carle Illinois Coll Med, Champaign, IL USA
[5] Univ Illinois, Canc Ctr Illinois, Urbana, IL USA
来源：
MOLECULAR THERAPY | 2019年 / 27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：
211
引用
收藏
页码：106 / 107
页数：2
相关论文
共 50 条
  • [1] AAV-mediated protein trans-splicing in the retina
    Tornabene, Patrizia
    Trapani, Ivana
    Dell'Aquila, Fabio
    Marrocco, Elena
    Gesualdo, Carlo
    Rossi, Settimio
    Giaquinto, Laura
    Albert, Silvia
    Hoyng, Carel B.
    Polishchuk, Elena
    Cremers, Frans P.
    Surace, Enrico Maria
    Simonelli, Francesca
    De Matteis, Antonella
    Polishchuk, Roman
    Auricchio, Alberto
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2019, 60 (09)
  • [2] AAV-Mediated Protein Trans-Splicing in the Retina
    Tornabene, Patrizia
    Trapani, Ivana
    Minopoli, Renato
    Centrulo, Miriam
    Lupo, Mariangela
    de Simone, Sonia
    Tiberi, Paola
    Dell'Aquila, Fabio
    Marrocco, Elena
    Iodice, Carolina
    Gesualdo, Carlo
    Rossi, Settimio
    Giaquinto, Laura
    Albert, Silvia
    Hoyng, Carl B.
    Polishchuk, Elena
    Cremers, Frans P. M.
    Surace, Enrico M.
    Simonelli, Francesca
    De Matteis, Maria A.
    Polishchuk, Roman
    Auricchio, Alberto
    MOLECULAR THERAPY, 2019, 27 (04) : 91 - 91
  • [3] AAV-mediated protein trans-splicing in the retina
    Tornabene, P.
    Trapani, I.
    Toriello, E.
    Minopoli, R.
    Centrulo, M.
    Lupo, M.
    de Simone, S.
    Tiberi, P.
    Dell'Aquila, F.
    Iodice, C.
    Albert, S.
    Hoyng, C. B.
    Cremers, F. P. M.
    Gesualdo, C.
    Simonelli, F.
    Auricchio, A.
    HUMAN GENE THERAPY, 2018, 29 (12) : A80 - A80
  • [4] AAV-mediated protein trans-splicing and genome editing for therapy of Haemophilia A
    Lyubenova, H.
    Minopoli, R.
    Llado, M.
    Nusco, E.
    Auricchio, A.
    HUMAN GENE THERAPY, 2019, 30 (11) : A83 - A83
  • [5] AAV-intein mediated Factor VIII trans-splicing for gene therapy of Haemophilia A
    Esposito, F.
    Lyubenova, H.
    Tornabene, P.
    Auricchio, S.
    Nusco, E.
    Merlin, S.
    Olgasi, C.
    Follenzi, A.
    Auricchio, A.
    HUMAN GENE THERAPY, 2021, 32 (19-20) : A15 - A15
  • [6] AAV Intein-Mediated Factor VIII Trans-Splicing for Gene Therapy of Haemophilia A
    Esposito, Federica
    Lyubenova, Hristiana
    Tornabene, Patrizia
    Auricchio, Stefano
    Nusco, Edoardo
    Olgasi, Cristina
    Follenzi, Antonia
    Auricchio, Alberto
    MOLECULAR THERAPY, 2021, 29 (04) : 169 - 169
  • [7] Intein-Mediated Protein Trans-Splicing in Hemophilia A
    Lyubenova, Hristiana
    Esposito, Federica
    Tornabene, Patrizia
    Minopoli, Renato
    Nusco, Edoardo
    Auricchio, Alberto
    MOLECULAR THERAPY, 2020, 28 (04) : 169 - 169
  • [8] Large therapeutic gene delivery with dual-AAV vectors: enhancing intein protein trans-splicing systems
    Ferreira, M. V.
    Almeida, A. I.
    Fernandes, S.
    Coroadinha, A. S.
    HUMAN GENE THERAPY, 2022, 33 (23-24) : A38 - A39
  • [9] Inclusion of a Degron Reduces Levels of Undesired Inteins after AAV-Mediated Protein Trans-Splicing in the Retina
    Tornabene, Patrizia
    Trapani, Ivana
    Centrulo, Miriam
    Marrocco, Elena
    Minopoli, Renato
    Lupo, Mariangela
    Iodice, Carolina
    Gesualdo, Carlo
    Simonelli, Francesca
    Surace, Enrico Maria
    Auricchio, Alberto
    MOLECULAR THERAPY, 2021, 29 (04) : 125 - 125
  • [10] Inclusion of a degron reduces levels of undesired inteins after AAV-mediated protein trans-splicing in the retina
    Tornabene, Patrizia
    Trapani, Ivana
    Centrulo, Miriam
    Marrocco, Elena
    Minopoli, Renato
    Lupo, Mariangela
    Iodice, Carolina
    Gesualdo, Carlo
    Simonelli, Francesca
    Surace, Enrico M.
    Auricchio, Alberto
    MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2021, 23 : 448 - 459
12345