Base editing HbS to HbG-Makassar improves hemoglobin function supporting its use in sickle cell disease.
Nat Commun 2025
Publications
Our research contributions to the scientific community
Impact of hydroxycarbamide treatment on the whole-blood transcriptome in sickle cell disease.
Br J Haematol 2025
Dual α-globin-truncated erythropoietin receptor knockin restores hemoglobin production in α-thalassemia-derived erythroid cells.
Cell Rep 2025
Enhancement of erythropoietic output by Cas9-mediated insertion of a natural variant in haematopoietic stem and progenitor cells.
Nat Biomed Eng 2024
Concerns With Statistical Analysis of 2016 CDC Opioid Guideline and Sickle Cell Outcomes.
JAMA Intern Med 2024
A miniaturized wash-free microfluidic assay for electrical impedance-based assessment of red blood cell-mediated microvascular occlusion.
Biosens Bioelectron 2024
Can we use biomarkers to identify those at risk of acute pain from sickle cell disease?
Expert Rev Hematol 2024
Gene Therapies for Hemoglobinopathies: Promises and Challenges.
Hemoglobin 2024
Oxygen gradient ektacytometry-derived biomarkers are associated with acute complications in sickle cell disease.
Blood Adv 2024
iCLOTS: open-source, artificial intelligence-enabled software for analyses of blood cells in microfluidic and microscopy-based assays.
Nat Commun 2023
Genetic regulation of fetal hemoglobin across global populations.
medRxiv 2023
Africa must participate in finding a gene therapy cure for sickle-cell disease.
Nat Med 2022
Sickle Cell Disease Pathophysiology and Related Molecular and Biophysical Biomarkers.
Hematol Oncol Clin North Am 2022
Comprehensive analysis and accurate quantification of unintended large gene modifications induced by CRISPR-Cas9 gene editing.
Sci Adv 2022
Validation of single-gene noninvasive prenatal testing for sickle cell disease.
Am J Hematol 2022
Clonal Hematopoiesis and the Risk of Hematologic Malignancies after Curative Therapies for Sickle Cell Disease.
J Clin Med 2022
Comment on: Oxygen gradient ektacytometry does not predict pain in children with sickle cell anaemia.
Br J Haematol 2022
Burden of central nervous system complications in sickle cell disease: A systematic review and meta-analysis.
Pediatr Blood Cancer 2022
OcclusionChip: A functional microcapillary occlusion assay complementary to ektacytometry for detection of small-fraction red blood cells with abnormal deformability.
Front Physiol 2022
Comparisons of oxygen gradient ektacytometry parameters between sickle cell patients with or without α-thalassaemia.
Br J Haematol 2021
A polygenic score for acute vaso-occlusive pain in pediatric sickle cell disease.
Blood Adv 2021
Effects of Genotypes and Treatment on Oxygenscan Parameters in Sickle Cell Disease.
Cells 2021
Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells.
Nat Med 2021
Sequencing of 53,831 diverse genomes from the NHLBI TOPMed Program.
Nature 2021
Oxygen gradient ektacytometry-derived biomarkers are associated with vaso-occlusive crises and correlate with treatment response in sickle cell disease.
Am J Hematol 2021
Methodological aspects of oxygen gradient ektacytometry in sickle cell disease: Effects of sample storage on outcome parameters in distinct patient subgroups.
Clin Hemorheol Microcirc 2021
Concurrent Assessment of Deformability and Adhesiveness of Sickle Red Blood Cells by Measuring Perfusion of an Adhesive Artificial Microvascular Network.
Front Physiol 2021
Rheological Impact of GBT1118 Cessation in a Sickle Mouse Model.
Front Physiol 2021
Loss-of-function genomic variants highlight potential therapeutic targets for cardiovascular disease.
Nat Commun 2020
A novel algorithm comprehensively characterizes human RH genes using whole-genome sequencing data.
Blood Adv 2020
The vaso-occlusive pain crisis in sickle cell disease: Definition, pathophysiology, and management.
Eur J Haematol 2020
Pediatric to Adult Transition in Sickle Cell Disease: Survey Results from Young Adult Patients.
Acta Haematol 2020
Methodological aspects of the oxygenscan in sickle cell disease: A need for standardization.
Am J Hematol 2020
Blood rheology biomarkers in sickle cell disease.
Exp Biol Med (Maywood) 2020
End points for sickle cell disease clinical trials: patient-reported outcomes, pain, and the brain.
Blood Adv 2019
Characterization of Sickling During Controlled Automated Deoxygenation with Oxygen Gradient Ektacytometry.
J Vis Exp 2019
A novel high-throughput molecular counting method with single base-pair resolution enables accurate single-gene NIPT.
Sci Rep 2019
Highly efficient editing of the β-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease.
Nucleic Acids Res 2019
Spontaneous healing of avascular necrosis of the femoral head in sickle cell disease.
Am J Hematol 2019
Metformin induces FOXO3-dependent fetal hemoglobin production in human primary erythroid cells.
Blood 2018
A Retrospective Analysis of Sociodemographic and Hematologic Characteristics Associated With Achieving Optimal Hydroxyurea Therapy in Children With Sickle Cell Disease.
J Pediatr Hematol Oncol 2018
Whole-exome sequencing of sickle cell disease patients with hyperhemolysis syndrome suggests a role for rare variation in disease predisposition.
Transfusion 2018
Fetal haemoglobin induction in sickle cell disease.
Br J Haematol 2018
Genetic modifiers of severity in sickle cell disease.
Clin Hemorheol Microcirc 2018
Echocardiography Differentiates Lethally Irradiated Whole-Body From Partial-Body Exposed Rats.
Front Cardiovasc Med 2018
p53 Nongenotoxic Activation and mTORC1 Inhibition Lead to Effective Combination for Neuroblastoma Therapy.
Clin Cancer Res 2017
The role of BCL11A and HMIP-2 polymorphisms on endogenous and hydroxyurea induced levels of fetal hemoglobin in sickle cell anemia patients from southern Brazil.
Blood Cells Mol Dis 2016
Original Research: Use of hydroxyurea and phlebotomy in pediatric patients with hemoglobin SC disease.
Exp Biol Med (Maywood) 2016
Inhibition of Listeria monocytogenes in deli-style turkey breast formulated with cultured celery powder and/or cultured sugar-vinegar blend during storage at 4°c.
J Food Prot 2014
Hepatitis C and neutropenia.
Curr Opin Hematol 2014
Whole exome sequencing identifies novel genes for fetal hemoglobin response to hydroxyurea in children with sickle cell anemia.
PLoS One 2014
Transcranial Doppler velocity and brain MRI/MRA changes in children with sickle cell anemia on chronic transfusions to prevent primary stroke.
Pediatr Blood Cancer 2013
Genetic modifiers of sickle cell anemia in the BABY HUG cohort: influence on laboratory and clinical phenotypes.
Am J Hematol 2013
Genetic mapping and exome sequencing identify 2 mutations associated with stroke protection in pediatric patients with sickle cell anemia.
Blood 2013
Severe neutropenia in patients with chronic hepatitis C: a benign condition.
Acta Haematol 2013
Dantrolene sodium for the treatment of aldesleukin-induced rigors in a melanoma patient.
Ann Pharmacother 2012
Improving gastric transit, gastrointestinal persistence and therapeutic efficacy of the probiotic strain Bifidobacterium breve UCC2003.
Microbiology (Reading) 2007
Heterologous expression of BetL, a betaine uptake system, enhances the stress tolerance of Lactobacillus salivarius UCC118.
Appl Environ Microbiol 2006