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A review on disease modifying pharmacologic therapies for sickle cell disease.
Ann Hematol
January 2025
Department of Internal Medicine, Section of Hematology/Oncology, University of Missouri-Kansas City, Kansas City, MO, 64108, USA.
Sickle cell disease (SCD) is an inherited hematologic disease caused by sickle hemoglobin as the predominant RBC hemoglobin or by sickle hemoglobin in combination with other abnormal β-hemoglobin variants like HbC, HbD and others. Sickling of erythrocytes under deoxygenated conditions is the basis of inflammatory and thrombotic cascades which result in multiple serious complications, leading to early morbidity and mortality. While HLA-matched allogeneic bone marrow transplantation is potentially curative, it has considerable limitations due to potential severe toxicities.
View Article and Find Full Text PDFAutologous CD19-Targeting CAR T Cells in a Patient With Refractory Juvenile Dermatomyositis.
Arthritis Rheumatol
October 2024
Bambino Gesù Children's Hospital, IRCCS, Catholic University of the Sacred Heart, Rome, Italy.
Article Synopsis
- The study focuses on the safety and effectiveness of CD19-targeting CAR T cells in a 12-year-old boy with resistant juvenile dermatomyositis (JDM).
- The patient underwent a single CAR T cell infusion after intensive lymphodepletion and showed significant immune response with complete B cell depletion and a mild side effect profile.
- Remarkably, he experienced sustained clinical improvements over eight months post-infusion without needing ongoing immunosuppressive therapy.
Advancing rare disease treatment: EMA's decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation.
Gene Ther
July 2024
Orphan Medicines Office, European Medicines Agency, Domenico Scarlattilaan 6, 1083 HS, Amsterdam, The Netherlands.
Adoptive cell therapy (ACT), particularly chimeric antigen receptor (CAR)-T cell therapy, has emerged as a promising approach for targeting and treating rare oncological conditions. The orphan medicinal product designation by the European Union (EU) plays a crucial role in promoting development of medicines for rare conditions according to the EU Orphan Regulation.This regulatory landscape analysis examines the evolution, regulatory challenges, and clinical outcomes of genetically engineered ACT, with a focus on CAR-T cell therapies, based on the European Medicines Agency's Committee for Orphan Medicinal Products review of applications evaluated for orphan designation and maintenance of the status over a 10-year period.
View Article and Find Full Text PDFHypertrophic Preconditioning Attenuates Myocardial Ischaemia-Reperfusion Injury by Modulating SIRT3-SOD2-mROS-Dependent Autophagy.
Cell Prolif
July 2021
Department of Cardiology, Shanghai Institute of Cardiovascular Diseases, Zhongshan Hospital, Fudan University, Shanghai, China.
Background: Ischaemic preconditioning elicited by brief periods of coronary occlusion and reperfusion protects the heart from a subsequent prolonged ischaemic insult. Here, we test the hypothesis that short-term non-ischaemic stimulation of hypertrophy renders the heart resistant to subsequent ischaemic injury.
Methods And Results: Transient transverse aortic constriction (TAC) was performed for 3 days in mice and then withdrawn for 4 days by aortic debanding, followed by subsequent exposure to myocardial ischaemia-reperfusion (I/R) injury.
Long noncoding RNA SOX2OT maintains the stemness of pancreatic cancer cells by regulating DEK via interacting with miR-200a/141.
Eur Rev Med Pharmacol Sci
December 2020
Department of Hematology, the First Hospital of Jilin University, Changchun, China.
The article "Long noncoding RNA SOX2OT maintains the stemness of pancreatic cancer cells by regulating DEK via interacting with miR-200a/141, by C.-S. Liu, Q.
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