Polynucleotides and uses in the treatment of prader-willi syndrome (PWS) and / or prader-willi like syndrome

An isolated polynucleotide with a specific sequence interacts with target mRNAs to regulate gene expression in PWS, effectively addressing the inefficiencies of current treatments by restoring normal DGKK gene expression levels.

WO2025061686A1 Publication Date: 2025-03-27UNIVERSITY OF LORRAINE +1
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Patent Information

Application Number
PCT/EP2024/075936
Authority / Receiving Office
WO · WO
Patent Type
Applications
Current Assignee / Owner
Priority Date
2023-09-22
Filing Date
2024-09-17
Publication Date
2025-03-27

AI Technical Summary

Technical Problem

Current treatments for Prader-Willi Syndrome (PWS) are inefficient, often resulting in genome-wide epigenetic effects and only minor reactivation of SNORD116 gene expression, which is insufficient to effectively address the symptoms of the syndrome.

Method used

An isolated polynucleotide with a sequence having at least 90% sequence identity with GGXaGGCAXbXcCCXdXeGGAAAGG (SEQ ID NO 1), where Xa, Xb, Xc, Xd, and Xe are independently T or U, is used to interact with target mRNAs produced by the DGKK, NLGN3, and RSBN1 L genes, thereby regulating their expression and restoring normal levels of the DGKK gene.

Benefits of technology

The polynucleotide effectively regulates the expression of genes dysregulated in PWS, restoring normal expression levels of the DGKK gene, which is crucial for treating PWS and Prader Willi-like syndromes.

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Abstract

The invention relates to an isolated polynucleotide comprising a sequence having at least 90% sequence identity with sequence GGXaGGCAXbXcCCXdXeGGAAAGG (SEQ ID NO 1), wherein Xa, Xb, Xc, Xd and Xe are independently T or U, and its use in the treatment of Prader-Willi Syndrome and / or Prader Willi-like syndromes. The invention also relates to pharmaceutical compositions an isolated polynucleotide comprising a sequence having at least 90% sequence identity with sequence GGXaGGCAXbXcCCXdXeGGAAAGG (SEQ ID NO 1), wherein Xa, Xb, Xc, Xd and Xe are independently T or U and its use in the prevention and / or treatment of Prader-Willi Syndrome and / or Prader Willi-like syndromes. The present invention finds applications in the therapeutic and diagnostic medical technical fields.
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