Antisense Oligonucleotide Mediated Increase in OPA1 Improves Mitochondrial Function in Fibroblasts Derived from Patients with Autosomal Dominant Optic Atrophy (ADOA)

被引：0
|
作者：
Venkatesh, Aditya [1 ]
Ali, Syed [1 ]
Oh, Raymond S. [1 ]
Sonntag, Donna [1 ]
Li, Zhiyu [2 ]
McKenty, Taylor [1 ]
Slipp, Brittany A. [1 ]
Hufnagel, Robert B. [2 ]
Hoger, Jeffrey H. [1 ]
Liau, Gene [1 ]
机构：
[1] Stoke Therapeut, Bedford, MA USA
[2] NIH, Bldg 10, Bethesda, MD 20892 USA
来源：
MOLECULAR THERAPY | 2021年 / 29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：
443
引用
收藏
页码：215 / 215
页数：1
相关论文
共 50 条
  • [1] 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)
  • [2] Antisense Oligonucleotide STK-002 Increases OPA1 in Retina and Improves Mitochondrial Function in Autosomal Dominant Optic Atrophy Cells
    Venkatesh, Aditya
    McKenty, Taylor
    Ali, Syed
    Sonntag, Donna
    Ravipaty, Shobha
    Cui, Yanyan
    Slate, Deirdre
    Lin, Qian
    Christiansen, Anne
    Jacobson, Sarah
    Kach, Jacob
    Lim, Kian Huat
    Srinivasan, Vaishnavi
    Zinshteyn, Boris
    Aznarez, Isabel
    Huryn, Laryssa A.
    Li, Zhiyu
    Hufnagel, Robert B.
    Liau, Gene
    Anderson, Karen
    Hoger, Jeff
    NUCLEIC ACID THERAPEUTICS, 2024, 34 (05) : 221 - 233
  • [3] 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)
  • [4] 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
  • [5] 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
  • [6] 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
  • [7] OPA1 deletions account for 26% of cases with autosomal dominant optic atrophy (ADOA, Kjer disease)
    Zanlonghi, Xavier
    Lenaers, Guy
    Charif, Majida
    Barrucand, Anne
    Guichet, Agnes
    Reynier, Pascal
    Desquiret-Dumas, Valerie
    Amati-Bonneau, Patrizia
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2023, 64 (08)
  • [8] Optic disc morphology of patients with OPA1 autosomal dominant optic atrophy
    Votruba, M
    Thiselton, D
    Bhattacharya, SS
    BRITISH JOURNAL OF OPHTHALMOLOGY, 2003, 87 (01) : 48 - 53
  • [9] OPA1 mutations and mitochondrial DNA haplotypes in autosomal dominant optic atrophy
    Han, J
    Thompson-Lowrey, AJ
    Reiss, A
    Mayorov, V
    Jlia, H
    Biousse, V
    Newman, NJ
    Brown, MD
    GENETICS IN MEDICINE, 2006, 8 (04) : 217 - 225
  • [10] New insights into OPA1 autosomal dominant optic atrophy
    Whitehead, Michael
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2023, 64 (08)
12345