AAV-CRISPR/Cas9 Gene Editing is Therapeutic in a Novel, Humanized Mouse Model of GUCY2D-Associated Cone Rod Dystrophy (CORD6)

被引：0
|
作者：
Mellen, Russell W. [1 ]
McCullough, K. Tyler [1 ]
Fajardo, Diego [1 ]
Calabro, Kaitlyn [1 ]
Crosson, Sean [1 ]
Xu, Emily [1 ]
Boye, Sanford L. [1 ]
Boye, Shannon E. [2 ]
机构：
[1] Univ Florida, Dept Pediat, Gainesville, FL USA
[2] Univ Florida, Dept Pediat, Div Cellular & Mol Therapy, Gainesville, FL USA
来源：
MOLECULAR THERAPY | 2022年 / 30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：
1216
引用
收藏
页码：566 / 566
页数：1
相关论文
共 29 条
  • [1] An AAV-CRISPR/Cas9 Gene Editing Approach for GUCY2D-Associated Cone Rod Dystrophy (CORD6)
    Mellen, R. W.
    McCullough, K. T.
    Boye, S. L.
    Fajardo, D.
    Strang, C. E.
    Witherspoon, C. D.
    Hill, J. L.
    Gloskowski, S. W.
    Dass, A.
    Gamlin, P. D.
    Maeder, M. L.
    Boye, S. E.
    MOLECULAR THERAPY, 2019, 27 (04) : 429 - 429
  • [2] An AAV-CRISPR/Cas9 gene editing approach for GUCY2D-associated cone rod dystrophy (CORD6)
    Mccullough, Kevin
    Boye, Sanford L.
    Fajardo, Diego
    Strang, Christianne E.
    Witherspoon, Douglas C.
    Gloskowski, Sebastian
    Dass, Abhishek
    Gamlin, Paul D.
    Maeder, Morgan
    Boye, Shannon Elizabeth
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2018, 59 (09)
  • [3] Development of a Novel Cone-Rod Dystrophy 6 (CORD6) Mouse Model to Assess AAV-CRISPR/Cas9-Based Therapies
    Mellen, Russell W.
    Boye, Sanford L.
    McCullough, K. Tyler
    Fajardo, Diego
    Crosson, Sean
    Calabro, Kaitlyn
    Dizhoor, Alex M.
    Boye, Shannon E.
    MOLECULAR THERAPY, 2020, 28 (04) : 267 - 267
  • [4] Systemic Gene Editing for Muscular Dystrophy Using AAV-CRISPR/Cas9
    Bengtsson, Niclas E.
    Hall, John K.
    Chamberlain, Jeffrey S.
    MOLECULAR THERAPY, 2017, 25 (05) : 16 - 16
  • [5] Somatic Gene Editing of GUCY2D by AAV-CRISPR/Cas9 Alters Retinal Structure and Function in Mouse and Macaque
    McCullough, K. Tyler
    Boye, Sanford L.
    Fajardo, Diego
    Calabro, Kaitlyn
    Peterson, James J.
    Strang, Christianne E.
    Chakraborty, Dibyendu
    Gloskowski, Sebastian
    Haskett, Scott
    Samuelsson, Steven
    Jiang, Haiyan
    Witherspoon, C. Douglas
    Gamlin, Paul D.
    Maeder, Morgan L.
    Boye, Shannon E.
    HUMAN GENE THERAPY, 2019, 30 (05) : 571 - 589
  • [6] Application of a CRISPR/Cas9 Gene Editing Therapy in a Novel Humanized Dystrophic Mouse Model of Duchenne Muscular Dystrophy
    Young, Courtney S.
    Quinonez, Marbella
    Mokhonova, Ekaterina
    Cannon, Stephen C.
    Pyle, April D.
    Spencer, Melissa J.
    MOLECULAR THERAPY, 2017, 25 (05) : 193 - 193
  • [7] Application of a CRISPR/Cas9 gene editing therapy in a novel humanized dystrophic mouse model of Duchenne muscular dystrophy
    Young, C.
    Mokhonova, E.
    Quinonez, M.
    Pyle, A.
    Spencer, M.
    NEUROMUSCULAR DISORDERS, 2017, 27 : S186 - S187
  • [8] A humanized knockin mouse model of Duchenne muscular dystrophy and its correction by CRISPR-Cas9 therapeutic gene editing
    Zhang, Yu
    Li, Hui
    Nishiyama, Takahiko
    McAnally, John R.
    Sanchez-Ortiz, Efrain
    Huang, Jian
    Mammen, Pradeep P. A.
    Bassel-Duby, Rhonda
    Olson, Eric N.
    MOLECULAR THERAPY-NUCLEIC ACIDS, 2022, 29 : 525 - 537
  • [9] A humanized knockin mouse model of Duchenne muscular dystrophy and its correction by CRISPR-Cas9 therapeutic gene editing
    Zhang, Yu
    Li, Hui
    Nishiyama, Takahiko
    McAnally, John R.
    Sanchez-Ortiz, Efrain
    Huang, Jian
    Mammen, Pradeep P. A.
    Bassel-Duby, Rhonda
    Olson, Eric N.
    MOLECULAR THERAPY-NUCLEIC ACIDS, 2022, 29 : 525 - 537
  • [10] CRISPR Cas9/gRNAs Selective Targeting of the GUCY2D Mutant Allele for Autosomal Dominant Cone-Rod Retinal Dystrophy
    Park-Windhol, Cindy
    Weingarden, Paul
    Takeuchi, Ryo
    Noma, Akiko
    Muraca, Maria L.
    Allocca, Mariacarmela
    Esteve-Rudd, Julian
    Lukason, Michael
    Neubauer, Jonas
    Dauletbekov, Daniyar
    Scharenberg, Andrew M.
    Fischer, M. Dominik
    Scaria, Abraham
    MOLECULAR THERAPY, 2019, 27 (04) : 263 - 263
123