Gene therapy of rare diseases as a milestone in medicine - overview of the field and report on initial experiences in Slovenia

Gene therapy of rare diseases as a milestone in medicine - overview of the field and report on initial experiences in Slovenia

Abstract Gene therapy has transitioned from a long-awaited promise to a clinical reality, offering transformative treatments for rare congenital diseases and certain cancers, which have a significant impact on patients’ lives. Current approaches focus on gene replacement therapy, either in vivo or e...

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Main Authors: Urh Grošelj, Marko Kavčič, Ana Drole Torkar, Jan Kafol, Duško Lainšček, Roman Jerala, Matjaž Sever, Samo Zver, Gregor Serša, Maja Čemažar, Primož Strojan, Aleš Grošelj, Mojca Žerjav Tanšek, Špela Miroševič, Simona Ivančan, Tomaž Prelog, David Gosar, Jasna Oražem Mrak, Matej Mlinarič, Sara Bertok, Jernej Kovač, Jana Kodrič, Saba Battelino, Marko Pokorn, Alojz Ihan, Janez Jazbec, Tadej Battelino, Damjan Osredkar
Format: Article
Language:English
Published: BMC 2025-06-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Gene therapy
Rare genetic diseases
Slovenia
CRISPR/Cas9
CAR-T cells
Cancer
Online Access:https://doi.org/10.1186/s13023-025-03828-8
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