Developing an AAV-Based Gene Replacement Therapy for Mitochondrial Alanyl-tRNA Synthetase 2 (AARS2) Leukodystrophy

被引:0
|
作者
Xing, Ruxiao [1 ]
Ren, Lingzhi [1 ]
Liu, Nan [1 ]
Zhou, Chen [1 ]
Liang, Jialing [1 ]
Wang, Jiaming [1 ,2 ]
Gao, Guangping [1 ,2 ]
Wang, Dan [1 ,3 ]
机构
[1] Univ Massachusetts, Chan Med Sch, Horae Gene Therapy Ctr, Worcester, MA 01605 USA
[2] Univ Massachusetts, Dept Microbiol & Physiol Syst, Chan Med Sch, Worcester, MA 01605 USA
[3] Univ Massachusetts, RNA Therapeut Inst, Chan Med Sch, Worcester, MA 01605 USA
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
36
引用
收藏
页码:20 / 20
页数:1
相关论文
共 50 条
  • [21] Developing an AAV-Based Gene Therapy for CSA Related Cockayne Syndrome
    Scholand, Aine
    Callahan, William
    Izzo, Cassandra
    King, Robert
    Sena-Esteves, Miguel
    Batista, Ana Rita
    MOLECULAR THERAPY, 2024, 32 (04) : 319 - 320
  • [22] Nrf2 dictates the neuronal survival and differentiation of embryonic zebrafish harboring compromised alanyl-tRNA synthetase
    Jin, Binbin
    Xie, Liqin
    Zhan, Dan
    Zhou, Luping
    Feng, Zhi
    He, Jiangyong
    Qin, Jie
    Zhao, Congjian
    Luo, Lingfei
    Li, Li
    DEVELOPMENT, 2022, 149 (17):
  • [23] Recessive Inheritance of a Rare Variant in the Nuclear Mitochondrial Gene for AARS2 in Late-Onset Dilated Cardiomyopathy
    Nielsen, Soren K.
    Hansen, Frederikke
    Schroder, Henrik Daa
    Wibrand, Flemming
    Gustafsson, Finn
    Mogensen, Jens
    CIRCULATION-GENOMIC AND PRECISION MEDICINE, 2020, 13 (05): : 560 - 562
  • [24] A Recurrent Loss-of-Function Alanyl-tRNA Synthetase (AARS) Mutation in Patients with Charcot-Marie-Tooth Disease Type 2N (CMT2N) (vol 33, pg 244, 2012)
    McLaughlin, Heather M.
    Sakaguchi, Reiko
    Giblin, William
    Wilson, Thomas E.
    Biesecker, Leslie
    Lupski, James R.
    Talbot, Kevin
    Vance, Jeffery M.
    Zuechner, Stephan
    Lee, Yi-Chung
    Kennerson, Marina
    Hou, Ya-Ming
    Nicholson, Garth
    Antonellis, Anthony
    HUMAN MUTATION, 2014, 35 (04) : 512 - 512
  • [25] Recombinant AAV-based gene therapy of phenylketonuria in the Pahenu2 missense mutant mouse
    Laipis, PJ
    Charron, CE
    Steele, HA
    Embury, JE
    Ross, K
    Knapp, JM
    Porvasnik, SL
    Alexander, JJ
    Reyes, L
    Zori, RT
    MOLECULAR THERAPY, 2003, 7 (05) : S391 - S392
  • [26] Pre-clinical efficacy testing of AAV-based gene therapy for KCNV2-deficiency
    Carvalho, Livia
    Rashwan, Rabab
    Lim, Xin Ru
    Brunet, Alicia
    Miller, Annie Laura
    Bhatt, Yashvi
    Fuller-Carter, Paula
    Hunt, David
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2023, 64 (08)
  • [27] AAV-based gene replacement therapy prevents and halts manifestation of abnormal neurological phenotypes in a novel mouse model of PMM2-CDG
    Zhong, Mian-ling
    Lai, Kent
    GENE THERAPY, 2025,
  • [28] Developing an AAV-Based Gene Therapy for MYBPC3 Mutation-Associated Hypertrophic Cardiomyopathy
    Salameh, Therese
    Xu, Hongbin
    Duan, Hong
    Sharma, Yogeshwar
    Yedlapudi, Deepthi
    Ren, Chao
    Cao, Jingsong
    Shi, Zhong-Dong
    MOLECULAR THERAPY, 2024, 32 (04) : 312 - 312
  • [29] Too Much or Too Little: AAV-Based Gene Replacement Therapy for SURF1-Related Leigh Syndrome
    Ling, Qinglan
    Rioux, Matthew
    Gray, Steven J.
    MOLECULAR THERAPY, 2023, 31 (04) : 341 - 341
  • [30] Evidence that the mitochondrial leucyl tRNA synthetase (LARS2) gene represents a novel type 2 diabetes susceptibility gene
    t Hart, LM
    Hansen, T
    Rietveld, I
    Dekker, JM
    Nijpels, G
    Janssen, GMC
    Arp, PA
    Uitterlinden, AG
    Jorgensen, T
    Borch-Johnsen, K
    Pols, HAP
    Pedersen, O
    van Duijn, CM
    Heine, RJ
    Maassen, JA
    DIABETES, 2005, 54 (06) : 1892 - 1895