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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A rare phenomenon known as acquired haemophilia is due to formation of antibodies against coagulation factors which can be idiopathic (mostly) or due to disseminated tuberculosis or paraneoplastic response or due to association with autoimmune disease, or recent pregnancy1. In acquired FVIII deficiency, haemorrhage tends to affect skin, muscle, soft tissue, and mucous membranes, rather than haemarthrosis2. The incidence of the acquired FVIII inhibitor is
Source: Transfusion and Apheresis Science - Category: Hematology Authors: Tags: Case Report Source Type: research
Publication date: Available online 21 May 2020Source: Multiple Sclerosis and Related DisordersAuthor(s): Elizabeth R. Comini-Frota, Angelo Pontes Freitas Campos, Antonio Pereira Gomes Neto, Paulo Pereira Christo
Source: Multiple Sclerosis and Related Disorders - Category: Neurology Source Type: research
In humans, maternal IgGs are transferred to the fetus from the second trimester of pregnancy onwards. The transplacental delivery of maternal IgG is mediated by its binding to the neonatal Fc receptor (FcRn) after endocytosis by the syncytiotrophoblast. IgGs present in the maternal milk are also transferred to the newborn through the digestive epithelium upon binding to the FcRn. Importantly, the binding of IgGs to the FcRn is also responsible for the recycling of circulating IgGs that confers them with a long half-life. Maternally delivered IgG provides passive immunity to the newborn, for instance by conferring protectiv...
Source: Frontiers in Immunology - Category: Allergy & Immunology Source Type: research
Acquired hemophilia A (AHA) is a rare autoimmune disease due to acquired IgG1-4 autoantibodies against coagulation factor VIII (FVIII). This causes patients, who have negative family and personal histories, to present with a sudden onset of heavy bleeding.1 –3 Although rare, the presence of antibodies against factors V and IX is possible.4
Source: Pediatrics and Neonatology - Category: Perinatology & Neonatology Authors: Tags: Letter to the Editor Source Type: research
CONCLUSIONS: Our findings suggest that a careful comprehensive geriatric assessment of acquired TTP patients is necessary. It is important to look for other autoimmune diseases and such age-related comorbidities as osteoporosis, arterial hypertension, ischemic heart disease and cerebrovascular disease. PMID: 32201091 [PubMed - as supplied by publisher]
Source: European Journal of Internal Medicine - Category: Internal Medicine Authors: Tags: Eur J Intern Med Source Type: research
CONCLUSIONS: The topics here discussed prove the undisputed role of mAbs as innovative biopharmaceuticals molecules and as vital components of targeted pharmacological therapies. PMID: 32072887 [PubMed - as supplied by publisher]
Source: Current Medicinal Chemistry - Category: Chemistry Authors: Tags: Curr Med Chem Source Type: research
Authors: Menegatti M, Biguzzi E, Peyvandi F Abstract Autoantibodies toward clotting factors may develop in people suffering from autoimmune or neoplastic diseases, after drug intake or even in subjects without apparent conditions. They are more commonly directed against factor VIII (FVIII) or von Willebrand factor leading to acquired hemophilia A or acquired von Willebrand syndrome, respectively. Rarely, autoantibodies develop against other clotting factors, such as fibrinogen, FII, FV, FVII, FX, FXI, and FXIII. The clinical picture of an acquired bleeding disorder includes a wide spectrum of clinical manifestation...
Source: Hematology ASH Education Program - Category: Hematology Tags: Hematology Am Soc Hematol Educ Program Source Type: research
We present a very rare case of adquired A-Hemophilia due to an abnormal paraprotein
Source: Clinical Lymphoma, Myeloma and Leukemia - Category: Hematology Authors: Source Type: research
Abstract Trials of antigen-specific tolerance have been undertaken in the clinic for over fifty years and the results of these antigen-specific clinical trials are described in this review. Antigen-specific tolerization of the immune system in protein replacement therapy for hemophilia A is an accepted treatment. Clinical trials are ongoing for autoimmune conditions such as type 1 diabetes, multiple sclerosis, neuromyelitis optica, and rheumatoid arthritis with various antigen-specific strategies. Trials for tolerization in celiac disease aim for antigen specific tolerance to gluten, an environmental trigger, whic...
Source: Current Opinion in Immunology - Category: Allergy & Immunology Authors: Tags: Curr Opin Immunol Source Type: research
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
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