AAV-mediated delivery of ABCA4 to the photoreceptors of Abca4-/- mice using an overlapping dual vector strategy

被引:0
作者
McClements, Michelle E. [1 ]
Barnard, Alun R. [1 ]
Singh, Mandeep S. [2 ]
Jiang, Zhichun [3 ]
Issa, Peter Charbel [4 ]
Radu, Roxana A. [3 ]
MacLaren, Robert E. [1 ,4 ]
机构
[1] Univ Oxford, Nuffield Dept Clin Neurosci Ophthalmol, Oxford, Oxon, England
[2] Johns Hopkins Univ, Baltimore, MD USA
[3] David Geffen Sch Med, Stein Eye Inst, Ophthalmol, Los Angeles, CA USA
[4] Oxford Eye Hosp, Oxford, England
关键词
D O I
暂无
中图分类号
R77 [眼科学];
学科分类号
100212 ;
摘要
4506
引用
收藏
页数:3
相关论文
共 50 条
  • [31] Using Oxford Nanopore long-range sequencing to phase ABCA4
    Mc Clinton, Benjamin
    Crinnion, Laura A.
    Ali, Manir
    Inglehearn, Chris
    Watson, Christopher M.
    Toomes, Carmel
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2021, 62 (08)
  • [32] Detection of the ABCA4 gene mutations using next-generation sequencing
    Oldak, Monika
    Sciezynska, Aneta
    Ambroziak, Anna Maria
    Korwin, Magdalena
    Ploski, Rafal
    Szaflik, Jacek P.
    Szaflik, Jerzy
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2014, 55 (13)
  • [33] RNAi-mediated lowering of transthyretin in liver leads to decreased accumulation of ocular A2E in Abca4-/- mice
    Ticau, Simina
    Keibler, Mark
    Liebovitz, Jacob
    Sridharan, Gautham
    Yao, Mark
    Cha, Diana
    Tymon, Roxanne
    Ho, Kaiya
    Adeduji, Olufemi
    Maddox, Callie
    Lambros, Mandy
    Vanlandingham, Phillip
    Farjo, Rafal
    Erbe, David
    Walshe, Tony
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2024, 65 (07)
  • [34] Oral Delivery of the P2Y12 Receptor Antagonist Ticagrelor Prevents Loss of Photoreceptors in an ABCA4-/- Mouse Model of Retinal Degeneration
    Lu, Wennan
    Campagno, Keith E.
    Tso, Huen-Yee
    Cenaj, Aurora
    Laties, Alan M.
    Carlsson, Leif G.
    Mitchell, Claire H.
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2019, 60 (08) : 3046 - 3053
  • [35] Enhanced expression of ABCA4 Stargardt protein mediated by adenoviral vectors in a simulated ocular environment
    Mallam, JN
    Hurwitz, MY
    Palmer, D
    Ng, P
    Hurwitz, RL
    MOLECULAR THERAPY, 2004, 9 : S52 - S52
  • [36] Proof-of-concept for multiple AON delivery by a single U7snRNA vector to restore splicing defects in ABCA4
    Suarez-Herrera, Nuria
    Riswick, Iris B.
    Vazquez-Dominguez, Irene
    Duijkers, Lonneke
    Karjosukarso, Dyah W.
    Piccolo, Davide
    Bauwens, Miriam
    De Baere, Elfride
    Cheetham, Michael E.
    Garanto, Alejandro
    Collin, Rob W. J.
    MOLECULAR THERAPY, 2024, 32 (03) : 837 - 851
  • [37] Lipopeptide-mediated Cas9 RNP delivery: A promising broad therapeutic strategy for safely removing deep-intronic variants in ABCA4
    Vazquez-Dominguez, Irene
    Oktem, Mert
    Winkelaar, Florian A.
    Nguyen, Thai Hoang
    Hoogendoorn, Anita D. M.
    Roschi, Eleonora
    Astuti, Galuh D. N.
    Timmermans, Raoul
    Suarez-Herrera, Nuria
    Bruno, Ilaria
    Ruiz-Llombart, Albert
    Brealey, Joseph
    de Jong, Olivier G.
    Collin, Rob W. J.
    Mastrobattista, Enrico
    Garanto, Alejandro
    MOLECULAR THERAPY NUCLEIC ACIDS, 2024, 35 (04):
  • [38] Single residue AAV capsid mutation improves transduction of photoreceptors in the Abca4−/− mouse and bipolar cells in the rd1 mouse and human retina ex vivo
    S R De Silva
    P Charbel Issa
    M S Singh
    D M Lipinski
    A O Barnea-Cramer
    N J Walker
    A R Barnard
    M W Hankins
    R E MacLaren
    Gene Therapy, 2016, 23 : 767 - 774
  • [39] Restoration of lysosomal pH in RPE cells from cultured human and ABCA4-/- mice:: Pharmacologic approaches and functional recovery
    Liu, Ji
    Lu, Wennan
    Reigada, David
    Nguyen, Jonathan
    Laties, Alan M.
    Mitchell, Claire H.
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2008, 49 (02) : 772 - 780
  • [40] ABCA4 gene replacement in a mouse model of Stargardt disease using a mammalian transposon system
    LeBlanc, Michelle
    Shukla, Shashank
    Rush, Kelsey A.
    Kopacz, Mitchell R.
    Hwang, Jungyeon
    Crowley, Michael J.
    Dutta, Subhadeep
    Vashishth, Ria
    Lu, Jonathan
    Nema, Sandeep
    Senn, Joe
    Higgins, Joseph J.
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2024, 65 (07)