WO2025037261 - METHODS OF PREVENTING OR TREATING LIVER DISEASE

National phase entry is expected:
Publication Number WO/2025/037261
Publication Date 20.02.2025
International Application No. PCT/IB2024/057917
International Filing Date 15.08.2024
Title **
[English] METHODS OF PREVENTING OR TREATING LIVER DISEASE
[French] MÉTHODES DE PRÉVENTION OU DE TRAITEMENT D'UNE MALADIE HÉPATIQUE
Applicants **
GLAXOSMITHKLINE INTELLECTUAL PROPERTY (NO.3) LIMITED
Inventors
LU, Qin
Priority Data
63/519,925   16.08.2023   US
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Quotation for National Phase entry

Country StagesTotal
China Filing, Examination, Granting2572
EPO Filing, Examination, Granting14225
Japan Filing, Examination, Granting2464
South Korea Filing, Examination, Granting2700
USA Filing, Examination, Granting6940
MasterCard Visa
Total: 28,901

The term for entry into the National Phase has expired. This quotation is for informational purposes only

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Abstract[English] Methods of preventing or treating liver disease in a subject characterised as having a PNPLA3- 148M protein variant are provided. The methods include administering to the subject an oligonucleotide or a DNA molecule encoding an oligonucleotide, wherein the oligonucleotide is capable of effecting ADAR-mediated editing of a polynucleotide encoding the PNPLA3-148M protein variant, and wherein the polynucleotide encoding the PNPLA3-148M protein variant undergoes ADAR-mediated editing at a codon encoding the methionine at position 148 such that the polynucleotide encodes a PNPLA3-148V protein variant. The disclosure also provides methods of editing a target ribonucleic acid (RNA) molecule encoding a PNPLA3-148M protein variant and methods of restoring the function of the PNPLA3 protein in a subject characterised as having the PNPLA3-148M protein variant.[French] L'invention concerne des méthodes de prévention ou de traitement d'une maladie hépatique chez un sujet caractérisées en ce qu'elles comprennent un variant protéique PNPLA3-148M. Les méthodes comprennent l'administration au sujet d'un oligonucléotide ou d'une molécule d'ADN codant pour un oligonucléotide, l'oligonucléotide étant capable d'effectuer une édition médiée par ADAR d'un polynucléotide codant pour le variant protéique PNPLA3-148M, et le polynucléotide codant pour le variant protéique PNPLA3-148M subissant une édition médiée par ADAR au niveau d'un codon codant pour la méthionine en position 148 de telle sorte que le polynucléotide code pour un variant protéique PNPLA3-148V. La divulgation concerne également des méthodes d'édition d'une molécule d'acide ribonucléique (ARN) cible codant pour un variant protéique PNPLA3-148M et des méthodes de restauration de la fonction de la protéine PNPLA3 chez un sujet caractérisées comme comprenant le variant protéique PNPLA3-148M.