Advances in gene therapy for the treatment of sickle cell anemia

Authors

  • Dania Patricia Alvarez Vázquez Department of Academic Unit of Health, Sciences, Universidad Autonoma de Guadalajara, Guadalajara, Jalisco, Mexico
  • Alejandro Fabricio Aguirre Barajas Department of Academic Unit of Health, Sciences, Universidad Autonoma de Guadalajara, Guadalajara, Jalisco, Mexico
  • Karla Reyes Chávez Department of Academic Unit of Health, Sciences, Universidad Autonoma de Guadalajara, Guadalajara, Jalisco, Mexico
  • Maricarmen Aguilar Hernández Department of Academic Unit of Health, Sciences, Universidad Autonoma de Guadalajara, Guadalajara, Jalisco, Mexico
  • Kemberly Noemi Zambrano Ledesma Department of Academic Unit of Health, Sciences, Universidad Autonoma de Guadalajara, Guadalajara, Jalisco, Mexico
  • Tane Del Río Barrera Department of Academic Unit of Health, Sciences, Universidad Autonoma de Guadalajara, Guadalajara, Jalisco, Mexico
  • Gerardo Garcia Santiago Department of Academic Unit of Health, Sciences, Universidad Autonoma de Guadalajara, Guadalajara, Jalisco, Mexico https://orcid.org/0009-0009-9135-400X
  • Alejandra Arias Castro Department of Academic Unit of Health, Sciences, Universidad Autonoma de Guadalajara, Guadalajara, Jalisco, Mexico

DOI:

https://doi.org/10.18203/2320-6012.ijrms20242691

Keywords:

Sickle cell anaemia, Gene therapy, Hemoglobin F, CRISPR-Cas9-gRNA-68, CD34 hematology

Abstract

Currently, sickle cell anemia is a β-hemoglobinopathy, meaning a genetic autosomal recessive disease, affecting 400,000 newborns worldwide. Approximately 5% of the global population are carriers of genes causing this pathology. Regarding treatment, there are medical and pharmacological therapies for the disease; however, the only known curative option is allogeneic hematopoietic stem cell transplantation. Nonetheless, there is a high rejection rate, compounded by the fact that it is only available to a limited number of patients. Now, there is a new alternative: gene therapy, which is based on the addition and modification of genes.

References

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Published

2024-09-20

How to Cite

Vázquez, D. P. A., Barajas, A. F. A., Chávez, K. R., Hernández, M. A., Ledesma, K. N. Z., Barrera, T. D. R., Garcia Santiago, G., & Castro, A. A. (2024). Advances in gene therapy for the treatment of sickle cell anemia. International Journal of Research in Medical Sciences, 12(10), 3952–3956. https://doi.org/10.18203/2320-6012.ijrms20242691

Issue

Vol. 12 No. 10 (2024): October 2024

Section

Review Articles