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Gene & Protein in Disease Sickle cell disease’s journey

The FDA-approved gene therapies – Casgevy, Lyfgenia, cell-anemia/symptoms-causes/syc-20355876 [Last accessed
and the lentiviral gene therapy, as already discussed above on 2024 Jun 29].
– have only demonstrated short-term results, and the FDA 4. Bender MA, Carlberg K. Sickle cell disease. In: Adam MP,
on its website has specified that “patients who received Feldman J, Mirzaa GM, Pagon RA, Wallace SE, Amemiya
Casgevy or Lyfgenia will be followed in a long-term study A, editors. GeneReviews®. Seattle, WA: University of
to evaluate each product’s safety and effectiveness.” Washington, Seattle; 1993-2025.

Acknowledgments 5. Serjeant GR. The natural history of sickle cell disease. Cold
Spring Harb Perspect Med. 2013;3:a011783.
None. doi: 10.1101/cshperspect.a011783
Funding 6. Grosse SD, Odame I, Atrash HK, Amendah DD, Piel FB,
Williams TN. Sickle cell disease in Africa: A neglected
None. cause of early childhood mortality. Am J Prev Med.
2011;41(6):S398-S405.
Conflict of interest
doi: 10.1016/j.amepre.2011.09.013
The authors declare they have no competing interests.
7. Inusa BPD, Hsu LL, Kohli N, et al. Sickle cell disease-genetics,
Author contributions pathophysiology, clinical presentation and treatment. Int J
Neonatal Screen. 2019;5(2):20.
Conceptualization: Malpe Surekha Bhat, Samira Bolo doi: 10.3390/ijns5020020
Formal analysis: Malpe Surekha Bhat, Tulika Mishra,
Deepti Mankar 8. US Centers for Disease Controls and Prevention (CDC).
Resources: Malpe Surekha Bhat, Tulika Mishra, Tiara Calvo Sickle Cell Disease. Available from: https://www.cdc.gov/
sickle-cell/data/index.html [Last accessed on 2024 May 15].
Leon
Supervision: Malpe Surekha Bhat, Abrar Khan 9. Ansari J, Moufarrej YE, Pawlinski R, Gavins FN. Sickle
Writing – original draft: Samira Bolo, Uzoamaka Eziri cell disease: A malady beyond a hemoglobin defect in
Writing – review & editing: Malpe Surekha Bhat, Abrar cerebrovascular disease. Expert Rev Hematol. 2018;11:45-55.
Khan, Frank Navarrete doi: 10.1080/17474086.2018.1407240

Ethics approval and consent to participate 10. Piel FB, Tatem AJ, Huang Z, Gupta S, Williams TN,
Weatherall DJ. Global migration and the changing
Not applicable. distribution of sickle haemoglobin: A quantitative study
of temporal trends between 1960 and 2000. Lancet Glob
Consent for publication Health. 2014;2:e80-e89.

Not applicable. doi: 10.1016/S2214-109X(13)70150-5
11. Rees DC, Williams TN, Gladwin MT. Sickle-cell disease.
Availability of data Lancet. 2010;376:2018-2031.
PubMed and NEJM journals were used as the main sources doi: 10.1016/S0140-6736(10)61029-X
to obtain information through literature searches. The 12. Steinberg MH, Sebastiani P. Genetic modifiers of sickle cell
corresponding author has a subscription with NEJM and disease. Am J Hematol. 2012;87:795-803.
backdated journals were used for literature search.
doi: 10.1002/ajh.23232
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Volume 4 Issue 1 (2025) 12 doi: 10.36922/gpd.4361