Patents Assigned to FONDAZIONE TELETHON ETS (50%)
  • POLYNUCLEOTIDES USEFUL FOR CORRECTING MUTATIONS IN THE RAG1 GENE
    Publication number: 20240425851
    Abstract: The present invention relates to an isolated polynucleotide comprising from 5? to 3?: a first homology region, a nucleotide sequence encoding a RAG1 polypeptide or a RAG1 polypeptide fragment, and a second homology region for use in treating a RAG-deficient immunodeficiency.
    Type: Application
    Filed: October 11, 2022
    Publication date: December 26, 2024
    Applicants: OSPEDALE SAN RAFFAELE S.R.L. (50%), FONDAZIONE TELETHON ETS (50%)
    Inventors: Anna Villa, Luigi Naldini, Samuele Ferrari, Maria Carmina Castiello, Simona Porcellini, Daniele Canarutto