Gene therapy results in long-term rescue of photoreceptor function in a mouse model for Bardet-Biedl Syndrome 1

被引：0
|
作者：
Freitas, M. M. Fernandes [4 ,5 ]
Hernandez-Hernandez, V. [1 ,2 ,3 ]
Pearson, R. A. [4 ,5 ]
Sidoli, C. [1 ]
Guillermin, O. [1 ]
Carreno, G. [1 ]
Cuevas, E. [1 ]
Smith, A. J. [4 ,5 ]
Ali, R. R. [4 ,5 ]
Beales, P. L. [1 ,3 ]
机构：
[1] Axovia Therapeut Inc, London, England
[2] Brunel Univ London, London, England
[3] UCL, Inst Child Hlth Great Ormond St, London, England
[4] UCL, Inst Ophthalmol, London, England
[5] Kings Coll London, London, England
来源：
HUMAN GENE THERAPY | 2021年 / 32卷 / 19-20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：
P068
引用
收藏
页码：A49 / A49
页数：1
相关论文
共 50 条
  • [41] Natural history study of visual function in patients with BBS1 and BBS10-related Bardet-Biedl syndrome
    Pechhacker, Monika Grudzinska
    Jacobson, Samuel G.
    Strubbe, Ine
    Duncan, Jacque L.
    Drack, Arlene V.
    Vincent, Andrea L.
    Aleman, Tomas
    Van Cauwenbergh, Caroline
    De Baere, Elfride
    Dollfus, Helene
    Goetz, Nathalie
    Leroy, Bart Peter
    Vincent, Ajoy
    Heon, Elise
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2020, 61 (07)
  • [42] Olfactory Cilia Defects From a Single Point Mutationin BBS1 Result in Impaired Odor Detection and Perceptionin a Bardet-Biedl Syndrome Mouse Model
    Xie, Chao
    Habif, Julia
    Uytingco, Cedric
    Ukhanov, Kirill
    Zhang, Lian
    Sheffield, Val
    Martens, Jeffrey
    CHEMICAL SENSES, 2019, 44 (07) : E48 - E48
  • [43] Creation and Characterization of a Bardet-Biedl Syndrome (BBS) Mouse Model Containing Mutations in Sdccag8 Using CRISPR/Cas9
    Reed, Michelle
    Jiang, Li
    Baehr, Wolfgang
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2016, 57 (12)
  • [44] Phenotypic expression of Bardet-Biedl syndrome in patients homozygous for the common M390R mutation in the BBS1 gene
    Cox, Kyle F.
    Kerr, Natalie C.
    Kedrov, Marina
    Nishimura, Darryl
    Jennings, Barbara J.
    Stone, Edwin M.
    Sheffield, Val C.
    Iannaccone, Alessandro
    VISION RESEARCH, 2012, 75 : 77 - 87
  • [45] The human FEM1B gene maps to chromosome 15q22 and is excluded as the gene for Bardet-Biedl syndrome, type 4
    Ventura-Holman, T
    Haider, NB
    Maher, JF
    AMERICAN JOURNAL OF THE MEDICAL SCIENCES, 2000, 319 (04): : 268 - 270
  • [46] TUDCA Slows Retinal Degeneration in Two Different Mouse Models of Retinitis Pigmentosa and Prevents Obesity in Bardet-Biedl Syndrome Type 1 Mice
    Drack, Arlene V.
    Dumitrescu, Alina V.
    Bhattarai, Sajag
    Gratie, Daniel
    Stone, Edwin M.
    Mullins, Robert
    Sheffield, Val C.
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2012, 53 (01) : 100 - 106
  • [47] Long-term vision rescue by human neural progenitors in a rat model of photoreceptor degeneration
    Wang, Shaomei
    Girman, Sergej
    Lu, Bin
    Bischoff, Nicholas
    Holmes, Toby
    Shearer, Rebecca
    Wright, Lynda S.
    Svendsen, Clive N.
    Gamm, David M.
    Lund, Raymond D.
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2008, 49 (07) : 3201 - 3206
  • [48] Long-term cure of a mouse model of erythropoietic protoporphyria by preselective gene therapy
    Pauliuk, R
    Bachelot, T
    Wise, R
    Mathews-Roth, MM
    Leboulch, P
    EXPERIMENTAL HEMATOLOGY, 1999, 27 (07) : 62 - 62
  • [49] Rescue of photoreceptor function by AAV-mediated gene transfer in a mouse model of inherited retinal degeneration
    Jomary, C
    Vincent, KA
    Grist, J
    Neal, MJ
    Jones, SE
    GENE THERAPY, 1997, 4 (07) : 683 - 690
  • [50] Rescue of photoreceptor function by AAV-mediated gene transfer in a mouse model of inherited retinal degeneration
    C Jomary
    KA Vincent
    J Grist
    MJ Neal
    SE Jones
    Gene Therapy, 1997, 4 : 683 - 690
12345