Researchers ready B cells for novel cell therapy

(Seattle Children's) Scientists at Seattle Children's Research Institute are paving the way to use gene-edited B cells -- a type of white blood cell in the immune system -- to treat a wide range of potential diseases that affect children, including hemophilia and other protein deficiency disorders, autoimmune diseases, and infectious diseases. If successful, their research would open the door to offering this experimental cell therapy as the first-of-its-kind in clinical trials at Seattle Children's in as soon as five years.
Source: EurekAlert! - Infectious and Emerging Diseases - Category: Infectious Diseases Source Type: news

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We report a 68-year-old Thai woman diagnosed and treated for bullous pemphigoid (BP) for 11 months who recently presented with a 3-day history of extensive hemorrhagic bullae and large intra-oral buccal hematoma. Laboratory investigations confirmed a prolonged activated partial thromboplastin time, a low factor VIII level, a high factor VIII inhibitor level, and elevated anti-BPAG180 and anti-BPAG230 titers, confirming the diagnosis of BP associated with AHA. Immunosuppressive therapy with systemic corticosteroids and cyclophosphamide combined with bypassing agents for bleeding control resulted in significant clinical impr...
Source: Case Reports in Dermatology - Category: Dermatology Source Type: research
Roser Calvo* Patient Safety, Safety Science, AstraZeneca Pharmaceuticals, Gaithersburg, MD, United States Immune-related hematological adverse events are amongst the rare but potentially life-threatening complications of immune checkpoint inhibitors. The spectrum of these toxicities is broadening as the number of patients exposed to these agents is increasing. Yet, they are still relatively unknown to many clinicians, possibly due to a lack of specific diagnostic criteria, which poses a challenge for their recognition and proper reporting, and partly due to their low incidence, often too low to be noted in most c...
Source: Frontiers in Pharmacology - Category: Drugs & Pharmacology Source Type: research
Reena Goswami1, Gayatri Subramanian2, Liliya Silayeva1, Isabelle Newkirk1, Deborah Doctor1, Karan Chawla2, Saurabh Chattopadhyay2, Dhyan Chandra3, Nageswararao Chilukuri1 and Venkaiah Betapudi1,4* 1Neuroscience Branch, Research Division, United States Army Medical Research Institute of Chemical Defense, Aberdeen, MD, United States 2Department of Medical Microbiology and Immunology, University of Toledo College of Medicine and Life Sciences, Toledo, OH, United States 3Roswell Park Comprehensive Cancer Center, Buffalo, NY, United States 4Department of Physiology and Biophysics, Case Western Reserve University, Clev...
Source: Frontiers in Oncology - Category: Cancer & Oncology Source Type: research
We report a rare case of a compartment syndrome of the forearm in a 30-year-old woman 2.5 months postpartum as the presentation of pregnancy-associated AHA.
Source: Blood Coagulation and Fibrinolysis - Category: Hematology Tags: CASE REPORT Source Type: research
CONCLUSIONS: Rituximab appears to be an effective and well-tolerated treatment for autoimmune acquired haemophilia. However, its place remains to be specified. PMID: 30904179 [PubMed - as supplied by publisher]
Source: Revue de Medecine Interne - Category: Internal Medicine Tags: Rev Med Interne Source Type: research
We report the case of a 78-year-old a male patient presenting with symptoms of a hematoma after a fall three days previously. He is medically followed for multiple myeloma and bullous pemphigoid. Laboratory investigations revealed isolated and recent increased of activated partial thromboplastin time (80,6/32,1s) and a markedly low FVIII activity (
Source: Annales de Biologie Clinique - Category: Biochemistry Authors: Tags: Ann Biol Clin (Paris) Source Type: research
Conclusion: Inhibitors were common among HA patients, and both severe HA and immune system challenges (surgery and trauma) are independent risk factors for inhibitor development.
Source: Nigerian Journal of Clinical Practice - Category: Rural Health Authors: Source Type: research
We report a 35-year-old postpartum female with acquired hemophilia A who initially presented with pleural hemorrhage. Diagnoses: In this patient activated prothrombin time (PT) and activated partial thromboplastin time (APTT) were found, and the factor VIII activity was 12.6%, furthermore Bethesda assay showed a FVIII antibody titer of 7.4 Bethesda units (BUs). Interventions: The treatment requires a 2-pronged approach: treatment of the bleeding and elimination of the inhibitor. Outcomes: After hemostatic agents were used and inhibitors were eradicated, the patient achieved complete remission without relapse. Les...
Source: Medicine - Category: Internal Medicine Tags: Research Article: Clinical Case Report Source Type: research
This article gives a comprehensive overview of the current knowledge about the pathophysiology, diagnosis, and treatment of AHA.
Source: Drugs - Category: Drugs & Pharmacology Source Type: research
We present the case of a patient affected by active MS who failed to achieve disease control with several disease-modifying drugs and was thereafter successfully treated with alemtuzumab, obtaining no evidence of disease activity and a high quality of life. Twenty months after the first infusion of alemtuzumab the patient developed acquired haemophilia A (AHA), a treatable but potentially lifethreatening condition that should be considered a possible SADs associated to this drug. In order to allow an early diagnosis and to prevent possible complications of AHA, routine coagulation tests (prothrombin time and activated part...
Source: Multiple Sclerosis and Related Disorders - Category: Neurology Source Type: research
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