A5030239988- Hemoglobinopathies and Related Disorders
- Hematopoietic Stem Cell Transplantation
- CRISPR and Genetic Engineering
- Transplantation: Methods and Outcomes
- Prenatal Screening and Diagnostics
- Pluripotent Stem Cells Research
- Advanced biosensing and bioanalysis techniques
- Virus-based gene therapy research
- Chromosomal and Genetic Variations
- Phagocytosis and Immune Regulation
- Acute Myeloid Leukemia Research
- Immunotherapy and Immune Responses
- Epigenetics and DNA Methylation
- Mosquito-borne diseases and control
- Erythrocyte Function and Pathophysiology
- T-cell and B-cell Immunology
- Polyomavirus and related diseases
- Mesenchymal stem cell research
Boston Children's Hospital
2021-2024
Harvard University
2021-2024
Boston Children's Museum
2022
George Washington University
2015
National Cancer Institute
2010
Hematopoietic stem cell transplant is curative of sickle disease (SCD) but limited by donor availability. Searches for 85 patients with SCD without matched sibling donors from 2009–2013 using modern techniques (allele‐level HLA matching unrelated and higher total nucleated doses umbilical cord blood (UCB)) showed potential match probabilities 20% 8/8 HLA‐matched donors, 84% 7/8 97% two 4‐6/6 UCBs suitable ex‐vivo expanded/double transplant. performed age 43 months would have a 90% chance...
Bronchiolitis obliterans syndrome (BOS) is a severe manifestation of chronic graft-versus-host disease (cGVHD) following hematopoietic cell transplantation (HCT). Montelukast interrupts cysteinyl leukotriene (CysLT) activity and may diminish the activation homing cells to bronchioles subsequent fibrosis. We performed prospective phase II trial test whether montelukast altered lung decline for patients with BOS after HCT. In this single-arm, open-label, multi-institutional study, primary...
Gene editing the BCL11A erythroid enhancer is a validated approach to fetal hemoglobin (HbF) induction for β-hemoglobinopathy therapy, though heterogeneity in edit allele distribution and HbF response may impact its safety efficacy. Here we compared combined CRISPR-Cas9 endonuclease of +58 +55 enhancers with leading gene modification approaches under clinical investigation. We found that targeting 3xNLS-SpCas9 two sgRNAs resulted superior induction, including engrafting cells from sickle...
In animal models, regulatory T cells (Treg) have been demonstrated to control autoimmune inflammation and prevent acute graft versus host disease (AGVHD), but the role of Treg in chronic GVHD (CGVHD) remains unknown. a natural history study CGVHD patients (NCI 04-C-0281), we assessed frequency phenotype FoxP3+T blood affected tissues. We used immunofluorescent staining for FoxP3 confocal microscopy assess tissue biopsies from with oral lichenoid cutaneous CGVHD. Although no sequential were...
Background: Bronchiolitis obliterans syndrome (BOS) is a severe manifestation of chronic graft-versus-host disease (cGVHD) following hematopoietic cell transplantation (HCT). Montelukast interrupts cysteinyl leukotriene activity and may diminish the activation homing cells to bronchioles subsequent fibrosis. Methods: We performed prospective phase II trial test whether montelukast altered lung decline for patients with BOS after HCT in single arm, open-label, multi-institutional study...