New antisense oligonucleotide therapies reach first base in ALS

被引：0
|
作者：
Elia R. Lopez
William F. Borschel
Bryan J. Traynor
机构：
[1] National Institutes of Health,Therapeutic Development Branch, National Center for Advancing Translational Sciences
[2] National Institutes of Health,Laboratory of Neurogenetics, National Institute on Aging
来源：
Nature Medicine | 2022年 / 28卷
关键词：
D O I：
暂无
中图分类号：
学科分类号：
摘要：
Two studies highlight the evolution of antisense oligonucleotide therapy for amyotrophic lateral sclerosis, offering hope for an effective treatment.
引用
收藏
页码：25 / 27
页数：2
相关论文
共 50 条
  • [31] Progress and prospects in antisense oligonucleotide-mediated exon skipping therapies for Duchenne muscular dystrophy
    Chwalenia, Katarzyna
    Wood, Matthew J. A.
    Roberts, Thomas C.
    JOURNAL OF MUSCLE RESEARCH AND CELL MOTILITY, 2025,
  • [32] Tofersen, a SOD1 Antisense Oligonucleotide in Participants with ALS - Results From a Multiple Dose Study
    Cudkowicz, Merit
    Miller, Timothy
    Shaw, Pamela
    Bennett, C. Frank
    Lane, Roger
    Chang, Ih
    Liu, Yingying
    Chen, Weiping
    Nestorov, Ivan
    Graham, Danielle
    Fanning, Laura
    McNeill, Manjit
    Fradette, Stephanie
    Ferguson, Toby
    NEUROLOGY, 2020, 94 (15)
  • [33] A new approach to preparation of antisense oligonucleotide samples with microextraction by packed sorbent
    Nuckowski, Lukasz
    Kaczmarkiewicz, Anna
    Studzinska, Sylwia
    Buszewski, Boguslaw
    ANALYST, 2019, 144 (15) : 4622 - 4632
  • [34] Ethylcellulose nanoparticles as a new "in vitro" transfection tool for antisense oligonucleotide delivery
    Leitner, S.
    Grijalvo, S.
    Solans, C.
    Eritja, R.
    Garcia-Celma, M. J.
    Caldero, G.
    CARBOHYDRATE POLYMERS, 2020, 229
  • [35] Macrophage inclusions in cerebrospinal fluid following treatment initiation with antisense oligonucleotide therapies in motor neuron diseases
    Vidovic, Maximilian
    Menschikowski, Mario
    Freigang, Maren
    Lapp, Hanna Sophie
    Guenther, Rene
    NEUROLOGICAL RESEARCH AND PRACTICE, 2024, 6 (01):
  • [36] Macrophage inclusions in cerebrospinal fluid following treatment initiation with antisense oligonucleotide therapies in motor neuron diseases
    Maximilian Vidovic
    Mario Menschikowski
    Maren Freigang
    Hanna Sophie Lapp
    René Günther
    Neurological Research and Practice, 6
  • [37] Inflammatory bowel disease: new therapies from antisense oligonucleotides
    Marafini, Irene
    Monteleone, Giovanni
    ANNALS OF MEDICINE, 2018, 50 (05) : 361 - 370
  • [38] Towards Personalized Allele-Specific Antisense Oligonucleotide Therapies for Toxic Gain-of-Function Neurodegenerative Diseases
    Helm, Jacob
    Schoels, Ludger
    Hauser, Stefan
    PHARMACEUTICS, 2022, 14 (08)
  • [39] A Renaissance for Antisense Oligonucleotide Drugs in Neurology Exon Skipping Breaks New Ground
    Yokota, Toshifumi
    Takeda, Shin'ichi
    Lu, Qi-Long
    Partridge, Terence A.
    Nakamura, Akinori
    Hoffman, Eric P.
    ARCHIVES OF NEUROLOGY, 2009, 66 (01) : 32 - 38
  • [40] NEW TWISTS ON OLIGONUCLEOTIDE STRUCTURE - INCORPORATING NOVEL SUGARS AND BACKBONES INTO ANTISENSE THERAPEUTICS
    GRIFFEY, RH
    CHEN, SM
    CUMMINS, L
    MOHAN, V
    SASMOR, H
    SANGHVI, Y
    TENG, K
    COOK, PD
    ABSTRACTS OF PAPERS OF THE AMERICAN CHEMICAL SOCIETY, 1994, 207 : 45 - CARB
12345