Current Gene TherapyCurrent Gene TherapyCurrent Gene Therapy1566-52321875-5631Bentham Science64389510.2174/1566523223666230320120839Life SciencesResearch ArticleGene Therapy for Rare Genetic DiseasesLundstromKennethinfo@benthamscience.netYannakiEvangeliainfo@benthamscience.netChouJaniceinfo@benthamscience.netSaadFawzyinfo@benthamscience.net, Pan TherapeuticsHematology Department, Hematopoietic Cell Transplantation Unit,, Gene and Cell Therapy Center, George Papanikolaou HospitalSection on Cellular Differentiation, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of HealthDepartment of Gene Therapy, Saad Pharmaceuticals010120242414507012025Copyright ©; 2024, Bentham Science Publishers2024Bentham Science Publishershttps://journals.eco-vector.com/1566-5232/article/view/643895Gene therapyrare genetic diseasesviral vectorsduchenne muscular dystrophythalassemiatype I glycogen storage diseases.[Lundstrom K. Gene therapy cargos based on viral vector delivery. Current Gene Therapy 2023; 23(2): 111-34.][Kommareddy S, Tiwari SB, Amiji MM. Long-circulating polymeric nanovectors for tumor-selective gene delivery. Technol Cancer Res Treat 2005; 4(6): 615-25.][Tomanin R, Scarpa M. Why do we need new gene therapy viral vectors? Characteristics, limitations and future perspectives of viral vector transduction. Current Gene Therapy 2004; 4(4): 357-72.][Saad FA, Saad JF, Siciliano G, Merlini L, Angelini C. Duchenne muscular dystrophy gene therapy. Current Gene Therapy In press][Chou JY, Mansfield BC. Gene therapy for type I glycogen storage diseases. Current Gene Therapy 2007; 7(2): 79-88.][Psatha N, Georgakopoulou A, Li C, et al. Enhanced HbF reactivation by multiplex mutagenesis of thalassemic CD34+ cells in vitro and in vivo. Blood 2021; 138(17): 1540-53.][Kloess S, Kretschmer A, Stahl L, Fricke S, Koehl U. CAR-expressing natural killer cells for cancer retargeting. Transfus Med Hemother 2019; 46(1): 4-13.][Li Y, Chen J, Tsai SQ, Cheng Y. Easy-Prime: A machine learning-based prime editor design tool. Genome Biol 2021; 22(1): 235.]