A vector and a pharmaceutical composition for reducing the expression of nkcc1 in a subject in need thereof, as well as a related therapeutic treatment method

A treatment method and subject technology, applied in the direction of introducing foreign genetic material using vectors, biochemical equipment and methods, pharmaceutical formulations, etc., can solve problems such as undesired excessive diuresis, impaired treatment compliance, ototoxicity, etc.
CN110799649APending Publication Date: 2020-02-14INST NAT DE LA SANTE & DE LA RECHERCHE MEDICALE (INSERM) +4
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Patent Information

Authority / Receiving Office
CN · China
Patent Type
Applications(China)
Current Assignee / Owner
INST NAT DE LA SANTE & DE LA RECHERCHE MEDICALE (INSERM)
Publication Date
2020-02-14

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Abstract

The invention relates to an RNA interference (RNAi) strategy based on the use of artificial microRNA (amiR) to reduce NKCC1 expression. In particular, the invention relates to a vector that achieves neuron-specific expression of specific amiR against NKCC1 by using a human Synapsin promoter to drive transgene expression.
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Description

technical field

[0001] The present invention relates to vectors, pharmaceutical compositions and methods for reducing NKCC1 expression in a subject in need thereof. Background technique

[0002] The Na+, K+, 2Cl- cotransporter (NKCC) encoded by SLC12A2 (NKCC1) belongs to the subfamily of cation chloride cotransporters (CCCs), which provide electrically neutral transport of sodium, potassium and chloride across the plasma membrane. Several lines of evidence suggest that NKCC1 is involved in the pathogenesis of various diseases by regulating the concentration of intracellular chloride. For example, recent studies have shown that the chloride cotransporter NKCC1 is overexpressed in the brains of mouse models of DS and patients with Down syndrome (Deidda et al., 2015; WO2015091857A1). Down syndrome (DS) is the most common cause of intellectual disability in children and adults. Down syndrome is a genetic disorder caused by the presence of all or part of the third copy of human...

Claims

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