Gergely Imre, Bertalan Takács, Erik Czipa, Andrea Bakné Drubi, Gábor Jaksa, Dóra Latinovics, Andrea Nagy, Réka Karkas, Liza Hudoba, Bálint Márk Vásárhelyi, Gabriella Pankotai-Bodó, András Blastyák, Zoltán Hegedűs, Péter Germán, Balázs Bálint, Khaldoon Sadiq Ahmed Abdullah, Anna Georgina Kopasz, Anita Kovács, László G Nagy, Farkas Sükösd, Lajos Pintér, Thomas Rülicke, Endre Barta, István Nagy, Lajos Haracska, Lajos Mátés
DNA transposon-based gene delivery vectors represent a promising new branch of randomly integrating vector development for gene therapy. For the side-by-side evaluation of the piggyBa c and Sleeping Beauty systems-the only DNA transposons currently employed in clinical trials-during therapeutic intervention, we treated the mouse model of tyrosinemia type I with liver-targeted gene delivery using both transposon vectors. For genome-wide mapping of transposon insertion sites we developed a new next-generation sequencing procedure called streptavidin-based enrichment sequencing, which allowed us to identify approximately one million integration sites for both systems...
June 8, 2023: Molecular Therapy. Methods & Clinical Development