For research use only. Not for therapeutic Use.
IONIS-MAPTRx (Cat No.: I045373) is an investigational antisense oligonucleotide designed to reduce the production of tau protein by targeting its mRNA (encoded by the MAPT gene) in the central nervous system. Excessive or abnormal tau accumulation is a hallmark of neurodegenerative diseases such as Alzheimer’s disease and frontotemporal dementia. By lowering tau levels, IONIS-MAPTRx aims to slow disease progression and neuronal damage. Administered intrathecally, it is currently being evaluated in clinical trials as a disease-modifying therapy for tauopathies.
CAS Number | 2857842-32-9 |
Purity | ≥95% |
Reference | [1]. Laurence Mignon, et al. Design of the First-in-Human Study of IONIS-MAPTRx, a Tau-lowering Antisense Oligonucleotide, in Patients With Alzheimer Disease (S2.006). Neurology Apr 2018, 90 (15 Supplement) S2.006. [2]. Rationale for and Development of IONIS-MAPTRx, the First Tau-lowering Antisense Oligonucleotide, in Patients with Mild AD [3]. DeVos SL, Miller RL, Schoch KM, et al. Tau reduction prevents neuronal loss and reverses pathological tau deposition and seeding in mice with tauopathy. Sci Transl Med. 2017;9(374):eaag0481. [4]. Methods for reducing tau expression. WO2023004390A1 |
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