A5069379795- Metabolism and Genetic Disorders
- Mitochondrial Function and Pathology
- Acute Myeloid Leukemia Research
- CAR-T cell therapy research
- Virus-based gene therapy research
- Muscle Physiology and Disorders
- ATP Synthase and ATPases Research
- Muscle and Compartmental Disorders
- Inflammatory Myopathies and Dermatomyositis
- Viral Infectious Diseases and Gene Expression in Insects
Edmond and Lily Safra Children's Hospital
2021-2024
Sheba Medical Center
2021-2024
Israel Medical Association
2023
Chimeric antigen receptor (CAR) T cells directed against CD19 (CART19) are effective in B-cell malignancies, but little is known about the molecular factors predicting clinical outcome of CART19 therapy. The increasingly recognized relevance epigenetic changes cancer immunology prompted us to determine impact DNA methylation profiles on course.We recruited 114 patients with comprising 77 acute lymphoblastic leukemia and 37 non-Hodgkin lymphoma who were treated cells. Using a comprehensive...
Patients with single large-scale mitochondrial DNA (mtDNA) deletion syndromes (SLSMDs) usually present multisystemic disease, either as Pearson syndrome in early childhood or Kearns-Sayre later life. No disease-modifying therapies exist for SLSMDs. We have developed a method to enrich hematopoietic cells exogenous mitochondria, and we treated six patients SLSMDs through compassionate use program. Autologous CD34 + were augmented maternally derived healthy technology termed augmentation...
CD28-based CD19 chimaeric antigen receptor-modified (CAR-)Tcells were recently FDA-approved for adult acute lymphoblastic leukaemia (ALL). We report long-term outcome of 37 children and young adults treated with autologous CAR-T cells. The complete remission rate was 86%, which 71% polymerase chain reaction (PCR) minimal residual disease (MRD)-negative, 14% MRD-negative by flow cytometry, PCR MRD-positive. 26 patients proceeded to subsequent haematopoietic stem cell transplant (HSCT). 11 had...
Pearson syndrome (PS) and Kearns-Sayre (KSS) are single large-scale mitochondrial DNA deletion (SLSMD) syndromes. PS is characterized by severe, transient childhood cytopenia, whereas KSS typically manifests later in life without hematologic abnormalities. Despite distinct clinical presentations, both share a common deletion. Recent observations suggest potential link between progression myeloid malignancy development, indicating that bone marrow failure (BMF) may be key aspect of pathology...