Doxzen K.W.Adair J.E.Bazzo Y.M.F.Bukini D.Cornetta K.Dalal V.Guerino-Cunha R.L.Hongeng S.Jotwani G.Kityo-Mutuluuza C.Lakshmanan K.Mahlangu J.Makani J.Mathews V.Ozelo M.C.Rangarajan S.Scholefield J.Júnior J.B.S.McCune J.M.Mahidol University2024-05-202024-05-202024-05-08Science translational medicine Vol.16 No.746 (2024) , eadn1902https://repository.li.mahidol.ac.th/handle/20.500.14594/98372Gene therapies are designed to address the root cause of disease. As scientific understanding of disease prevention, diagnosis, and treatment improves in tandem with technological innovation, gene therapies have the potential to become safe and effective treatment options for a wide range of genetic and nongenetic diseases. However, as the medical scope of gene therapies expands, consideration must be given to those who will benefit and what proactive steps must be taken to widen development and access potential, particularly in regions carrying a high disease burden.MedicineReviewSCOPUS10.1126/scitranslmed.adn19022-s2.0-851926339251946624238718130