Page 86 - GPD-4-1
P. 86
Gene & Protein in Disease Sickle cell disease’s journey
doi: 10.3324/haematol.2018.213462 doi: 10.1056/NEJMoa0904971
110. Ndefo UA, Maxwell AE, Nguyen H, Chiobi TL. 116. Hsieh MM, Fitzhugh CD, Tisdale JF. Allogeneic
Pharmacological management of sickle cell disease. P T. hematopoietic stem cell transplantation for sickle cell
2008;33(4):238-243. disease: The time is now. Blood. 2011;118(5):1197-1207.
111. Eastep TG, Kendsersky RM, Zook J, Moore A. Penicillin doi: 10.1182/blood-2011-01-332510
prophylaxis in patients with sickle cell disease beyond age
5 years. J Pediatr Pharmacol Ther. 2023;28(6):519-523. 117. Saraf SL, Oh AL, Patel PR, et al. Nonmyeloablative stem cell
transplantation with alemtuzumab/low-dose irradiation to
doi: 10.5863/1551-6776-28.6.519 cure and improve the quality of life of adults with sickle cell
112. Ashorobi D, Naha K, Bhatt R. Hematopoietic Stem Cell disease. Biol Blood Marrow Transplant. 2016;22(3):441-448.
Transplantation in Sickle Cell Disease. In: StatPearls; 2023. doi: 10.1016/j.bbmt.2015.08.036
Available from: https://www.ncbi.nlm.nih.gov/books/
NBK538515 [Last accessed on 2024 Nov 07]. 118. Fliesler N. Sickle cell Gene Therapy and Boosting Fetal
Hemoglobin: A 75-Year History. Boston Children’s Hospital;
113. Leonard A, Tisdale JF. Stem cell transplantation in sickle cell 2023. Available from: https://answers.childrenshospital.
disease: Therapeutic potential and challenges faced. Expert org/sickle-cell-fetal-hemoglobin-timeline [Last accessed on
Rev Hematol. 2018;11(7):547-565. 2024 Apr 06].
doi: 10.1080/17474086.2018.1486703 119. Platt OS, Brambilla DJ, Rosse WF, et al. Mortality in sickle
114. FDA News Release. FDA Approves First Gene Therapies to cell disease. Life expectancy and risk factors for early death.
Treat Patients with Sickle Cell Disease; 2023. Available from: N Engl J Med. 1994;330:1639.
https://www.fda.gov/news-events/press-announcements/ doi: 10.1056/NEJM199406093302303
fda-approves-first-gene-therapies-treat-patients-sickle-cell-
disease [Last accessed on 2024 Nov 06]. 120. Lanzkron S, Carroll CP, Haywood C Jr. Mortality rates and
age at death from sickle cell disease: U.S., 1979-2005. Public
115. Hsieh MM, Kang EM, Fitzhugh CD, et al. Allogeneic
hematopoietic stem-cell transplantation for sickle cell Health Rep. 2013;128:110-116.
disease. N Engl J Med. 2009;361(24):2309-2317. doi: 10.1177/003335491312800206
Volume 4 Issue 1 (2025) 17 doi: 10.36922/gpd.4361

