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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.

Pending Publication Date: 2020-02-14
INST NAT DE LA SANTE & DE LA RECHERCHE MEDICALE (INSERM) +4
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  • Abstract
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Problems solved by technology

However, some data suggest the need for lifelong treatment with bumetanide, potentially causing undesired excessive diuresis (which can impair treatment adherence) and the many associated side effects of long-term electrolyte imbalance
In addition, systemic treatment with bumetanide also blocks NKCC1 activity in organs other than the brain, which may cause ototoxicity (Ben-Ari et al., 2016)

Method used

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  • 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 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 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

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Embodiment Construction

[0004] The inventors have developed an RNA interference (RNAi) strategy based on reducing NKCC1 expression using an artificial microRNA (amiR). By replacing the 21-22 nucleotide antisense of a naturally occurring primary microRNA (pri-miRNA) scaffold (such as mouse microRNA155; GenBank accession number: NR_029565.1) with an antisense targeting sequence against hNKCC1 Artificial microRNAs are constructed using a sense targeting sequence (the so-called guide strand). In particular, the inventors have designed a system that achieves neuron-specific expression of a specific amiR against NKCC1 by using the human synaptophysin promoter to drive transgene expression.

[0005] Therefore, in a first aspect, the present invention relates to a vector for use in a method of treatment for reducing the expression of NKCC1 in a subject in need thereof, the vector comprising a polynucleotide encoding an amiR capable of reducing the expression of NKCC1, wherein the amiR comprises a polynucleot...

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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.

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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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): C12N15/115C12N15/113
CPCC12N15/113C12N2310/141C12N2310/531A61K45/06C12N15/86
Inventor 费代里科·明戈齐朱塞佩·龙齐蒂安德烈亚·孔泰斯塔比莱劳拉·坎切达
Owner INST NAT DE LA SANTE & DE LA RECHERCHE MEDICALE (INSERM)
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