Gene Therapy for Rare Genetic Diseases
- Authors: Lundstrom K.1, Yannaki E.2, Chou J.3, Saad F.4
 - 
							Affiliations: 
							
- , Pan Therapeutics
 - Hematology Department, Hematopoietic Cell Transplantation Unit,, Gene and Cell Therapy Center, George Papanikolaou Hospital
 - Section on Cellular Differentiation, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health
 - Department of Gene Therapy, Saad Pharmaceuticals
 
 - Issue: Vol 24, No 1 (2024)
 - Pages: 4-5
 - Section: Life Sciences
 - URL: https://vietnamjournal.ru/1566-5232/article/view/643895
 - DOI: https://doi.org/10.2174/1566523223666230320120839
 - ID: 643895
 
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About the authors
Kenneth Lundstrom
, Pan Therapeutics
														Email: info@benthamscience.net
				                					                																			                												                														
Evangelia Yannaki
Hematology Department, Hematopoietic Cell Transplantation Unit,, Gene and Cell Therapy Center, George Papanikolaou Hospital
														Email: info@benthamscience.net
				                					                																			                												                														
Janice Chou
Section on Cellular Differentiation, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health
														Email: info@benthamscience.net
				                					                																			                												                														
Fawzy Saad
Department of Gene Therapy, Saad Pharmaceuticals
							Author for correspondence.
							Email: info@benthamscience.net
				                					                																			                												                														
References
- 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.
 
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