Pecker L, Roth M, Landman S, Cunningham L, Silver E, Manwani D
Ann Pediatr Child Health. 2024; 3(1).
PMID: 39712473
PMC: 11661846.
Zakieh A, Mercure-Corriveau N, Lanzkron S, Feng X, Vozniak S, Crowe E
Transfusion. 2024; 64(8):1509-1519.
PMID: 39003570
PMC: 11316647.
DOI: 10.1111/trf.17924.
Hulbert M, King A, Shenoy S
Hematology Am Soc Hematol Educ Program. 2022; 2022(1):277-282.
PMID: 36485131
PMC: 9820741.
DOI: 10.1182/hematology.2022000372.
Mekelenkamp H, van Zanten H, de Vries M, Lankester A, Smiers F
Front Pediatr. 2021; 9:690309.
PMID: 34485192
PMC: 8416427.
DOI: 10.3389/fped.2021.690309.
Yang P, Chou S, Li J, Hui W, Liu W, Sun N
Sci Adv. 2020; 6(43).
PMID: 33097539
PMC: 7608838.
DOI: 10.1126/sciadv.abb7107.
Discovery of Novel Fetal Hemoglobin Inducers through Small Chemical Library Screening.
Breveglieri G, Pacifico S, Zuccato C, Cosenza L, Sultan S, DAversa E
Int J Mol Sci. 2020; 21(19).
PMID: 33050052
PMC: 7582302.
DOI: 10.3390/ijms21197426.
Highly efficient editing of the β-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease.
Park S, Lee C, Dever D, Davis T, Camarena J, Srifa W
Nucleic Acids Res. 2019; 47(15):7955-7972.
PMID: 31147717
PMC: 6735704.
DOI: 10.1093/nar/gkz475.
Risk factors and outcomes according to age at transplantation with an HLA-identical sibling for sickle cell disease.
Cappelli B, Volt F, Tozatto-Maio K, Scigliuolo G, Ferster A, Dupont S
Haematologica. 2019; 104(12):e543-e546.
PMID: 31018975
PMC: 6959194.
DOI: 10.3324/haematol.2019.216788.
Sickle cell retinopathy. A focused review.
Abdalla Elsayed M, Mura M, Al Dhibi H, Schellini S, Malik R, Kozak I
Graefes Arch Clin Exp Ophthalmol. 2019; 257(7):1353-1364.
PMID: 30895451
DOI: 10.1007/s00417-019-04294-2.
Hydroxyurea Treated β-Thalassemia Children Demonstrate a Shift in Metabolism Towards Healthy Pattern.
Iqbal A, Ansari S, Parveen S, Khan I, Siddiqui A, Musharraf S
Sci Rep. 2018; 8(1):15152.
PMID: 30310134
PMC: 6182004.
DOI: 10.1038/s41598-018-33540-6.
Rare single gene disorders: estimating baseline prevalence and outcomes worldwide.
Blencowe H, Moorthie S, Petrou M, Hamamy H, Povey S, Bittles A
J Community Genet. 2018; 9(4):397-406.
PMID: 30109643
PMC: 6167259.
DOI: 10.1007/s12687-018-0376-2.
Bidirectional immune tolerance in nonmyeloablative MHC-mismatched BMT for murine β-thalassemia.
E S, Seth A, Vogel P, Sommers M, Ong T, Pillai A
Blood. 2017; 129(22):3017-3030.
PMID: 28351935
PMC: 5454334.
DOI: 10.1182/blood-2016-03-704387.
Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation.
Gluckman E, Cappelli B, Bernaudin F, Labopin M, Volt F, Carreras J
Blood. 2016; 129(11):1548-1556.
PMID: 27965196
PMC: 5356458.
DOI: 10.1182/blood-2016-10-745711.
Pediatric sickle cell disease: past successes and future challenges.
Meier E, Rampersad A
Pediatr Res. 2016; 81(1-2):249-258.
PMID: 27706129
DOI: 10.1038/pr.2016.204.
CRISPR-Cas9 technology and its application in haematological disorders.
Zhang H, McCarty N
Br J Haematol. 2016; 175(2):208-225.
PMID: 27619566
PMC: 5889078.
DOI: 10.1111/bjh.14297.
Optimizing autologous cell grafts to improve stem cell gene therapy.
Psatha N, Karponi G, Yannaki E
Exp Hematol. 2016; 44(7):528-39.
PMID: 27106799
PMC: 4914411.
DOI: 10.1016/j.exphem.2016.04.007.
The therapeutic potential of genome editing for β-thalassemia.
Glaser A, McColl B, Vadolas J
F1000Res. 2016; 4.
PMID: 26918126
PMC: 4753996.
DOI: 10.12688/f1000research.7087.1.
HLA-haploidentical blood or marrow transplantation with high-dose, post-transplantation cyclophosphamide.
Fuchs E
Bone Marrow Transplant. 2015; 50 Suppl 2:S31-6.
PMID: 26039204
PMC: 4634886.
DOI: 10.1038/bmt.2015.92.
Hematopoietic Stem Cell Transplantation in Adult Sickle Cell Disease: Problems and Solutions.
Ozdogu H, Boga C
Turk J Haematol. 2015; 32(3):195-205.
PMID: 25912490
PMC: 4563194.
DOI: 10.4274/tjh.2014.0311.
Recent trends in the gene therapy of β-thalassemia.
Finotti A, Breda L, Lederer C, Bianchi N, Zuccato C, Kleanthous M
J Blood Med. 2015; 6:69-85.
PMID: 25737641
PMC: 4342371.
DOI: 10.2147/JBM.S46256.