WO2025144082 - USH2A MINIPROTEIN FOR GENE THERAPY OF USHER SYNDROME TYPE II
National phase entry is expected:
Publication Number
WO/2025/144082
Publication Date
03.07.2025
International Application No.
PCT/RU2024/050227
International Filing Date
26.09.2024
Title **
[English]
USH2A MINIPROTEIN FOR GENE THERAPY OF USHER SYNDROME TYPE II
[French]
MINIPROTÉINE USH2A POUR LA THÉRAPIE GÉNIQUE DU SYNDROME DE USHER DE TYPE II
Applicants **
RETINAFUND
Inventors
MALOGOLOVKIN, Aleksandr Sergeevich
KOLESNIK, Valeriia Valerevna
KATORKIN, Sergei Aleksandrovich
POROZOV, Iurii Borisovich
NURTDINOV, Ruslan Faritovich
Priority Data
2023136407
31.12.2023
RU
Application details
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International Searching Authority |
Rospatent
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| Recordal of a Change of the Applicant's Name/Address |
Change of Applicant's Name and Address
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| Type of Assignment |
The Standard Agent's Assignment
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| Applicant's Legal Status |
Legal Entity
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| Entry into National Phase under |
Chapter I
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| Patent Delivery |
Send the Letters Patent by Courier
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| Translation |
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Quotation for National Phase entry
| Country | Stages | Total | |
|---|---|---|---|
| China | Filing, Examination, Granting | 2149 | |
| EPO | Filing, Examination, Granting | 11966 | |
| Japan | Filing, Examination, Granting | 2093 | |
| South Korea | Filing, Examination, Granting | 2004 | |
| USA | Filing, Examination, Granting | 5340 |
Total:
23,552
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Abstract[English]
The present invention relates to the field of biotechnology, namely to the USH2A miniprotein which, due to a small size enabling its delivery in viral vectors, retains LamGL, LamNT, EGF Lam, FN3 domains and PDZ-binding motif of a full-length protein, which are required for the USH2A protein function. Also disclosed is a nucleic acid encoding the USH2A miniprotein, an expression vector, and the use of said vector in the gene therapy of Usher syndrome type II. The invention can be effectively used for stable and high expression of the USH2A miniprotein.[French]
La présente invention concerne le domaine de la biotechnologie, à savoir la miniprotéine USH2A qui, en raison d'une petite taille permettant son administration dans des vecteurs viraux, retient des domaines LamGL, LamNT, EGF Lam et FN3 et un motif de liaison à PDZ d'une protéine pleine longueur, qui sont nécessaires pour la fonction protéique de USH2A. Est également divulgué un acide nucléique codant pour la miniprotéine USH2A, un vecteur d'expression et l'utilisation dudit vecteur dans la thérapie génique de syndrome de Usher de type II. L'invention peut être efficacement utilisée en vue d'une expression stable et élevée de la miniprotéine USH2A.