Sickle cell disease (SCD) has a widely variable clinical course; some of them are severely affected in childhood while in others major damage does not manifest until adulthood. There is no test that can be used to predict the disease course. The best predictor of SCD severity is a patient’s course to date [
]. Patients’ quality of life is low and life expectancy is relatively short, nearly 50 years, even newly developed treatments such as iron chelating and disease modifying agents. The only curative treatment option is allogeneic stem cell transplantation (Allo-SCT), but only one fifth of the patients have matched sibling donor. Even they have, there are several complications may develop during and after the transplantation [
- Romero Z.
- DeWitt M.
- Walters M.C.
Promise of gene therapy to treat sickle cell disease.
Expert Opin Biol Ther. 2018; 18: 1123-1136https://doi.org/10.1080/14712598.2018.1536119
]. This is why there is a safer, convenient and accessible treatments are needed for them. Gene therapy would be one of them.
- Benítez-Carabante M.I.
- Beléndez C.
- González-Vicent M.
- Alonso L.
- Uría-Oficialdegui M.L.
- Torrent M.
- et al.
Grupo Español de Trasplante de Médula Ósea en Niños (GETMON), Grupo Español de Trasplante Hematopoyético (GETH). Matched sibling donor stem cell transplantation for sickle cell disease: Results from the Spanish group for bone marrow transplantation in children.
Eur J Haematol. 2021; 106: 408-416https://doi.org/10.1111/ejh.13566
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- Promise of gene therapy to treat sickle cell disease.Expert Opin Biol Ther. 2018; 18: 1123-1136https://doi.org/10.1080/14712598.2018.1536119
- Grupo Español de Trasplante de Médula Ósea en Niños (GETMON), Grupo Español de Trasplante Hematopoyético (GETH). Matched sibling donor stem cell transplantation for sickle cell disease: Results from the Spanish group for bone marrow transplantation in children.Eur J Haematol. 2021; 106: 408-416https://doi.org/10.1111/ejh.13566
- Emerging therapies in sickle cell disease.Br J Haematol. 2020; 190: 149-172https://doi.org/10.1111/bjh.16504
- Hematopoietic stem cell gene-addition/editing therapy in sickle cell disease.Cells. 2022; 11: 1843https://doi.org/10.3390/cells11111843
- Manipulation of developmental gamma-globin gene expression: an approach for healing hemoglobinopathies.Mol Cell Biol. 2020; 41: e00253-20https://doi.org/10.1128/MCB.00253-20
- Gene therapy in a patient with sickle cell disease.N Engl J Med. 2017; 376: 848-855https://doi.org/10.1056/NEJMoa1609677
- Biologic and clinical efficacy of lentiglobin for sickle cell disease.New Engl J Med. 2022; 386: 617-628https://doi.org/10.1056/NEJMoa2117175
Olowoyeye A., Okwundu C.I. Gene therapy for sickle cell disease. Cochrane Database Syst Rev. 2018 Nov 16;11(11):CD007652. doi: 10.1002/14651858.CD007652.pub6. Update in: Cochrane Database Syst Rev. 2020; 11: CD007652.
- Gene therapy for sickle cell disease: moving from the bench to the bedside.Blood. 2021; 138: 932-941https://doi.org/10.1182/blood.2019003776
- Gene therapy for beta-hemoglobinopathies: milestones, new therapies and challenges.Mol Diagn Ther. 2019; 23: 173-186https://doi.org/10.1007/s40291-019-00383-4
- Gene therapy applications of non-human lentiviral vectors.Viruses. 2020; 12: 1106https://doi.org/10.3390/v12101106
- A universal approach to correct various hbb gene mutations in human stem cells for gene therapy of beta-thalassemia and sickle cell disease.Stem Cells Transl Med. 2018; 7: 87-97https://doi.org/10.1002/sctm.17-0066
- Foetal haemoglobin, erythrocytes containing foetal haemoglobin, and hematological features in congolese patients with sickle cell anaemia.Anemia. 2012; 2012105349https://doi.org/10.1155/2012/105349
- Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusion.Haematologica. 2018; 103: 778-786https://doi.org/10.3324/haematol.2017.184788
- Safety and efficacy of plerixafor dose escalation for the mobilization of CD34+ hematopoietic progenitor cells in patients with sickle cell disease: interim results.Haematologica. 2018; 103: 770-777https://doi.org/10.3324/haematol.2017.187047
- Parameters affecting successful stem cell collections for genetic therapies in sickle cell disease.Transfus Apher Sci. 2021; 60103059https://doi.org/10.1016/j.transci.2021.103059
- Autologous stem-cell-based gene therapy for inherited disorders: state of the art and perspectives.Front Pedia. 2019; 7: 443https://doi.org/10.3389/fped.2019.00443
- A systematic review and meta-analysis of gene therapy with hematopoietic stem and progenitor cells for monogenic disorders.Nat Commun. 2022; 13: 1315https://doi.org/10.1038/s41467-022-28762-2
- Myelodysplastic syndrome unrelated to lentiviral vector in a patient treated with gene therapy for sickle cell disease.Blood Adv. 2020; 4: 2058-2063https://doi.org/10.1182/bloodadvances.2019001330
Published online: February 24, 2023
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