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
2023
Thrombo-Inflammation in COVID-19 and Sickle Cell Disease: Two Faces of the Same Coin
Chiang K, Gupta A, Sundd P, Krishnamurti L. Thrombo-Inflammation in COVID-19 and Sickle Cell Disease: Two Faces of the Same Coin. Biomedicines 2023, 11: 338. PMID: 36830874, PMCID: PMC9953430, DOI: 10.3390/biomedicines11020338.Peer-Reviewed Original ResearchSickle cell diseaseVaso-occlusive crisisCell diseaseLipid mediatorsCOVID-19Severe SARS-CoV-2 infectionSARS-CoV-2 infectionSevere COVID-19Platelet-leukocyte adhesionAnti-thrombotic agentsThrombo-InflammationEndothelial injuryRespiratory infectionsSevere illnessClinical trialsThromboxane ATherapeutic strategiesTherapeutic targetPotential treatmentPlatelet activationComplement activationGreater riskClinical researchDiseaseEndothelial cells