Gene Therapy for Rare Genetic Diseases

Curr Gene Ther. 2024;24(1):4-5. doi: 10.2174/1566523223666230320120839.

¹ Pan Therapeutics, CH1095 Lutry, Switzerland.
² Hematology Department, Hematopoietic Cell Transplantation Unit, Gene and Cell Therapy Center, George Papanikolaou Hospital, 57010 Thessaloniki, Greece.
³ Section on Cellular Differentiation, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD 20892, USA.
⁴ Department of Gene Therapy, Saad Pharmaceuticals, Juhkentali 8, Tallinn 10145, Estonia.

No abstract available

Keywords: Gene therapy; duchenne muscular dystrophy; rare genetic diseases; thalassemia; type I glycogen storage diseases.; viral vectors.

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