2024
Zinc finger nuclease-mediated gene editing in hematopoietic stem cells results in reactivation of fetal hemoglobin in sickle cell disease
Lessard S, Rimmelé P, Ling H, Moran K, Vieira B, Lin Y, Rajani G, Hong V, Reik A, Boismenu R, Hsu B, Chen M, Cockroft B, Uchida N, Tisdale J, Alavi A, Krishnamurti L, Abedi M, Galeon I, Reiner D, Wang L, Ramezi A, Rendo P, Walters M, Levasseur D, Peters R, Harris T, Hicks A. Zinc finger nuclease-mediated gene editing in hematopoietic stem cells results in reactivation of fetal hemoglobin in sickle cell disease. Scientific Reports 2024, 14: 24298. PMID: 39414860, PMCID: PMC11484757, DOI: 10.1038/s41598-024-74716-7.Peer-Reviewed Original ResearchConceptsHematopoietic stem cellsSickle cell diseaseTreatment of sickle cell diseaseFetal hemoglobinCell therapyReactivation of fetal hemoglobinCell diseaseMonths of follow-upStem cellsReactivate fetal hemoglobinResults of preclinical studiesPotential treatmentEngraftment in vivoAutologous cell therapyNovel cell therapiesVaso-occlusive crisisIncreased total hemoglobinErythroid progenyHealthy donorsPreclinical studiesClinical developmentFollow-upErythroid enhancerBCL11A erythroid enhancerGATAA motifs
2014
Feasibility of a Newborn Screening and Follow-up Programme for Sickle Cell Disease among South Gujarat (India) Tribal Populations
Italia Y, Krishnamurti L, Mehta V, Raicha B, Italia K, Mehta P, Ghosh K, Colah R. Feasibility of a Newborn Screening and Follow-up Programme for Sickle Cell Disease among South Gujarat (India) Tribal Populations. Journal Of Medical Screening 2014, 22: 1-7. PMID: 25341880, DOI: 10.1177/0969141314557372.Peer-Reviewed Original ResearchConceptsSickle cell diseaseSCD babiesCell diseaseNewborn screeningSevere clinical complicationsHigh fetal hemoglobinNewborn screening programsSickle cell disordersClinical presentationTribal populationClinical complicationsNatural courseScreening programNewborn babiesCell disordersXmn I polymorphismBabiesDiseaseHome careFetal hemoglobinHigh-performance liquid chromatographyRegular monitoringScreeningPopulationΑ-thalassaemia