Antisense oligonucleotides for the treatment of neurological disorders
RNA editing oligonucleotides enhance KCC2 activity by deaminating specific adenosines in the SLC12A5 transcript, addressing the need for effective treatments for neurological disorders by restoring inhibitory signaling.
WO2026060374A2PCT designated stage Publication Date: 2026-03-19PROQR THERAPEUTICS II BV +1
Patent Information
- Application Number
- PCT/US2025/046398
- Authority / Receiving Office
- WO · WO
- Patent Type
- Applications
- Current Assignee / Owner
- Priority Date
- 2025-08-07
- Filing Date
- 2025-09-15
- Publication Date
- 2026-03-19
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Figure US2025046398_19032026_PF_FP_ABST
Abstract
The disclosure relates to the field of diseases caused by a lowered synaptic inhibition, preferably those that are caused by a diminished activity of the potassium (K) / chloride (Cl) Cotransporter 2 (KCC2). The disclosure involves oligonucleotides and the use thereof in RNA editing methods in targeting a variety of target adenosines in the human SLC12A5 transcript molecule that encodes KCC2. The transcript molecule is edited such that the resulting KCC2 protein has a gain-of-function and / or different function, for example reduced autoinhibition. The disclosure relates to oligonucleotides and their use in the treatment of neurodevelopment disorders, neuropsychiatric disorders, chronic pain disorders, and / or epilepsy.
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Citation Information
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