Japan's Astellas to Buy Audentes for $3 Bln in High-Priced Gene Therapy Bet Japan's Astellas to Buy Audentes for $3 Bln in High-Priced Gene Therapy Bet

Japan's Astellas Pharma Inc is buying U.S. drugmaker Audentes Therapeutics Inc for about $3 billion in cash, in a high-priced push to make genetic medicines a key area of growth.Reuters Health Information
Source: Medscape Allergy Headlines - Category: Allergy & Immunology Tags: Family Medicine/Primary Care News Source Type: news

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Source: Journal of Allergy and Clinical Immunology - Category: Allergy & Immunology Authors: Source Type: research
Severe combined immunodeficiency (SCID) is a heterogeneous group of rare disorders caused by mutations in different genes involved in the development and function of immune cells. Patients with SCID are usually highly susceptible to severe opportunistic infection, and, if untreated, die within 1 year. Since the first successful bone marrow transplants in X-linked SCID and Wiskott-Aldrich syndrome, hematopoietic stem cell (HSC) transplantation (HSCT) has become the standard treatment for most patients with SCID.
Source: Journal of Allergy and Clinical Immunology - Category: Allergy & Immunology Authors: Tags: Editorial Source Type: research
Purpose of review We briefly address the advances in genetics, pathophysiology, and phenotypes of chronic granulomatous disease (CGD). This is one of the most studied primary immunodeficiencies, which comprise mutations in genes encoding the different subunits of the NADPH oxidase system. Those mutations lead to defective reactive oxygen species production, and consequently a failure to eliminate pathogens. Recent findings Patients with CGD are susceptible to fungal, bacterial, and parasitic infections. Other symptoms, as systemic adverse effects to BCG vaccine and hyperinflammation, are also important clinical condit...
Source: Current Opinion in Pediatrics - Category: Pediatrics Tags: ALLERGY, IMMUNOLOGY AND RELATED DISORDERS: Edited by Jordan S. Orange Source Type: research
Purpose of review X-linked agammaglobulinaemia (XLA) is a congenital defect of development of B lymphocytes leading to agammaglobulinaemia. It was one of the first primary immunodeficiencies described, but treatment has remained relatively unchanged over the last 60 years. This summary aims to outline the current outcomes, treatments and future research areas for XLA. Recent findings Immunoglobulin therapy lacks IgA and IgM, placing patients at theoretical risk of experiencing recurrent respiratory tract infections and developing bronchiectasis despite best current therapy. Recent cohort studies from Italy and the USA...
Source: Current Opinion in Allergy and Clinical Immunology - Category: Allergy & Immunology Tags: PRIMARY IMMUNE DEFICIENCY DISEASE: Edited by Stephen Jolles and M. Teresa (Maite) de la Morena Source Type: research
Patients with T-cell immunodeficiencies are generally treated with allogeneic hematopoietic stem cell transplantation, but alternatives are needed for patients without matched donors. An innovative intrathymic gene therapy approach that directly targets the thymus might improve outcomes.
Source: Journal of Allergy and Clinical Immunology - Category: Allergy & Immunology Authors: Source Type: research
Intrathymic AAV-mediated gene therapy presents a novel therapeutic option for immunodeficient patients, promoting a rapid reconstitution of the thymic environment and subsequent T cell reconstitution.
Source: Journal of Allergy and Clinical Immunology - Category: Allergy & Immunology Authors: Source Type: research
Severe combined immunodeficiency (SCID) represents the most lethal form of primary immunodeficiency, with mortality rates of greater than 90% within the first year of life without treatment. Hematopoietic stem cell transplantation and gene therapy are the only curative treatments available, and the best-known prognostic factors for success are age at diagnosis, age at hematopoietic stem cell transplantation, and the comorbidities that develop in between. There are no evidence-based guidelines for standardized clinical care for patients with SCID during the time between diagnosis and definitive treatment, and we aim to gene...
Source: Journal of Allergy and Clinical Immunology - Category: Allergy & Immunology Authors: Tags: Rostrum Source Type: research
Patients are about to be enrolled in the first study to test a gene-editing technique known as CRISPR inside the body to try to cure an inherited form of blindness. People with the disease have normal eyes but lack a gene that converts light into signals to the brain that enable sight. The experimental treatment aims to supply kids and adults with a healthy version of the gene they lack, using a tool that cuts or “edits” DNA in a specific spot. It’s intended as a onetime treatment that permanently alters the person’s native DNA. Two companies, Editas Medicine and Allergan, will test this in up to 18...
Source: TIME: Health - Category: Consumer Health News Authors: Tags: Uncategorized onetime Research Source Type: news
Conclusions This review describes how leukocyte-heparanase can be a double-edged sword in tumor progression; it can enhance tumor immune surveillance and tumor cell clearance, but also promote tumor survival and growth. We also discuss the potential of using heparanase in leukocyte therapies against tumors, and the effects of heparanase inhibitors on tumor progression and immunity. We are just beginning to understand the influence of heparanase on a pro/anti-tumor immune response, and there are still many questions to answer. How do the pro/anti-tumorigenic effects of heparanase differ across different cancer types? Does...
Source: Frontiers in Oncology - Category: Cancer & Oncology Source Type: research
An effective epidermal barrier requires intercellular junctions including desmosomes and gap junctions (GJ). While desmosomes play a role in cell-cell adhesion, GJs facilitate small molecule transfer across cell membranes. GJs are composed of connexins (Cx), with Cx43 (encoded by GJA1) most abundantly expressed in skin. Severe dermatitis, multiple Allergies and Metabolic wasting (SAM) syndrome is a condition caused by biallelic mutations in DSG1. SAM syndrome can manifest with skin lesions reminiscent of Erythrokeratodermia Variabilis, a group of disorders associated with mutations in Cx-encoding genes which result in Cx43 dysregulation.
Source: Journal of Investigative Dermatology - Category: Dermatology Authors: Tags: Genetic Disease, Gene Regulation, and Gene Therapy Source Type: research
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