Models of Autosomal Dominant Optic Atrophy (ADOA) using OPA1 haploinsufficient iPSCs and response to Targeted Augmentation of Nuclear Gene Output (TANGO) Antisense Oligonucleotides (ASOs) Treatment

被引：0
|
作者：
Oh, Raymond [1 ]
Slipp, Brittany [1 ]
Venkatesh, Aditya [1 ]
Ali, Syed [1 ]
Li, Zhiyu [1 ]
Hufnagel, Robert B. [1 ]
Aznarez, Isabel [1 ]
Liau, Gene [1 ]
Hoger, Jeff [1 ]
机构：
[1] Stoke Therapeut Inc, Bedford, MA USA
来源：
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE | 2022年 / 63卷 / 07期
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：
1895
引用
收藏
页数：3
相关论文
共 10 条
  • [1] Antisense oligonucleotide mediated increase of OPA1 expression using TANGO technology for the treatment of autosomal dominant optic atrophy
    Venkatesh, Aditya
    Li, Zhiyu
    Christiansen, Anne
    Lim, Kian Huat
    Kach, Jacob
    Hufnagel, Robert
    Aznarez, Isabel
    Liau, Gene
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2020, 61 (07)
  • [2] Antisense Oligonucleotide Mediated Increase of OPA1 Expression Using TANGO Technology for the Treatment of Autosomal Dominant Optic Atrophy
    Venkatesh, Aditya
    Li, Zhiyu
    Ali, Syed
    Christiansen, Anne
    Lim, Kian Huat
    Kach, Jacob
    Hufnagel, Robert B.
    Hoger, Jeffery H.
    Aznarez, Isabel
    Liau, Gene
    MOLECULAR THERAPY, 2020, 28 (04) : 112 - 112
  • [3] Clinical findings in patients with autosomal dominant optic atrophy (adOA) and OPA1 gene mutations.
    Kellner, U
    Pesch, UE
    Wissinger, B
    Jandeck, C
    Kraus, H
    Foerster, MH
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2001, 42 (04) : S78 - S78
  • [4] cDNA-based screening and analysis of the OPA1 gene in patients with autosomal dominant optic atrophy (adOA)
    Schimpf, S
    Schaich, S
    Wissinger, B
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2005, 46
  • [5] Analysis of opa1 isoforms expression and apoptosis regulation in autosomal dominant optic atrophy (ADOA) patients with mutations in the opal gene
    Formichi, Patrizia
    Radi, Elena
    Giorgi, Eleonora
    Gallus, Gian Nicola
    Brunetti, Jlenia
    Battisti, Carla
    Rufa, Alessandra
    Dotti, Maria Teresa
    Franceschini, Rossella
    Bracci, Luisa
    Federico, Antonio
    JOURNAL OF THE NEUROLOGICAL SCIENCES, 2015, 351 (1-2) : 99 - 108
  • [6] Antisense oligonucleotide mediated increase in OPA1 improves mitochondrial function in fibroblasts derived from patients with autosomal dominant optic atrophy (ADOA)
    Venkatesh, Aditya
    Ali, Syed
    Oh, Raymond S.
    Sonntag, Donna
    Li, Zhiyu
    McKenty, Taylor
    Slim, Brittany A.
    Hufnagel, Robert B.
    Hoger, Jeffrey
    Liau, Gene
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2021, 62 (08)
  • [7] Antisense Oligonucleotide Mediated Increase in OPA1 Improves Mitochondrial Function in Fibroblasts Derived from Patients with Autosomal Dominant Optic Atrophy (ADOA)
    Venkatesh, Aditya
    Ali, Syed
    Oh, Raymond S.
    Sonntag, Donna
    Li, Zhiyu
    McKenty, Taylor
    Slipp, Brittany A.
    Hufnagel, Robert B.
    Hoger, Jeffrey H.
    Liau, Gene
    MOLECULAR THERAPY, 2021, 29 (04) : 215 - 215
  • [8] Autosomal dominant optic atrophy: A novel treatment for OPA1 splice defects using U1 snRNA adaption
    Jueschke, Christoph
    Klopstock, Thomas
    Catarino, Claudia B.
    Owczarek-Lipska, Marta
    Wissinger, Bernd
    Neidhardt, John
    MOLECULAR THERAPY-NUCLEIC ACIDS, 2021, 26 : 1186 - 1197
  • [9] STK-002, an Antisense Oligonucleotide (ASO) for the Treatment of Autosomal Dominant Optic Atrophy (ADOA), Localizes to Retinal Ganglion Cells (RGC) and Upregulates OPA-1 Protein Expression After Intravitreal Administration to Non-Human Primates (NHP)
    Venkatesh, Aditya
    McKenty, Taylor
    Slate, Deidre
    Lin, Qian
    Ravipaty, Shobha
    Jacobson, Sarah
    Christiansen, Anne
    Anderson, Karen
    Benmohamed, Radhia
    Hoger, Jeff
    Liau, Gene
    MOLECULAR THERAPY, 2022, 30 (04) : 9 - 9
  • [10] STK-002, an Antisense Oligonucleotide (ASO) for the Treatment of Autosomal Dominant Optic Atrophy (ADOA), is Taken Up by Retinal Ganglion Cells (RGC) and Upregulates OPA-1 Protein Expression After Intravitreal Administration to Non-human Primates (NHPs)
    Anderson, Karen
    Venkatesh, Aditya
    McKenty, Taylor
    Slate, Deidre
    Lin, Qian
    Ravipaty, Shobha
    Jacobson, Sarah
    Christiansen, Anne
    Benmohamed, Radhia
    Hoger, Jeff
    Liau, Gene
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2022, 63 (07)
1