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Therapy: Gene Therapy
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Total 130 results found since Jan 2013.
News at a glance: Muscular dystrophy therapy, lab-grown chicken, and humans ’ toll on wildlife
BIOMEDICINE
Muscular dystrophy therapy approved
The U.S. Food and Drug Administration has approved the first gene therapy for Duchenne muscular dystrophy (DMD), a genetic disease that cripples boys and usually results in death by age 30. The treatment from Sarepta Therapeutics introduces a short version of the gene for dystrophin, a crucial muscle protein, which is mutated in patients with DMD. A one-time intravenous infusion of a virus delivers the functioning “microdystrophin” gene into patients’ muscle cells. The 22 June approval is only for boys 4 to 5 years old, a group that appeared likely to ben...
Source: Science of Aging Knowledge Environment - June 29, 2023 Category: Geriatrics Source Type: research
Intellia Shares Jump as Gene Therapy Trial Gets US Regulator Nod Intellia Shares Jump as Gene Therapy Trial Gets US Regulator Nod
Shares of gene editing firm Intellia Therapeutics soared 10% in early trade on Thursday after the US health regulator cleared its application to begin a mid-stage trial for its gene therapy NTLA-2002.Reuters Health Information
Source: Medscape Allergy Headlines - March 4, 2023 Category: Allergy & Immunology Tags: Pathology & Lab Medicine News Source Type: news
Gene therapy for SCID, now up to 3!
Since 2000, gene therapy has become an option to treat severe combined immunodeficiency (SCID), a rare but lethal condition characterized by fully defective T lymphocyte differentiation caused by pathogenic variants in least 18 genes. The current treatment by allogeneic hematopoietic stem cell transplantation (HSCT) is effective but carries the risk of graft-versus-host disease. This treatment is based on ex vivo gene transfer by using retroviral vector (initially gammaretrovirus, now lentivirus) to transduce the patient bone marrow CD34 cells.
Source: Journal of Allergy and Clinical Immunology - February 22, 2023 Category: Allergy & Immunology Authors: Alain Fischer, B énédicte Neven Tags: Editorial Source Type: research
Gene therapy of SCID, now up to 3 !
Source: Journal of Allergy and Clinical Immunology - February 22, 2023 Category: Allergy & Immunology Authors: Alain Fischer, B énédicte Neven Source Type: research
Mechanism of Kruppel-Like Factor 4 in Pyroptosis of Nasal Mucosal Epithelial Cells in Mice With Allergic Rhinitis
CONCLUSION: KLF4 bound to the NLRP3 promoter and promoted pyroptosis of NEpCs in AR mice via activating NLRP3.PMID:36799547 | DOI:10.1177/19458924221148568
Source: American Journal of Rhinology and Allergy - February 17, 2023 Category: ENT & OMF Authors: Jiaoli Yao Qingfeng Kong Yin Wang Yanting Zhang Qinxue Wang Source Type: research
Gastrointestinal and Hepatic Manifestations of Chronic Granulomatous Disease
J Allergy Clin Immunol Pract. 2023 Jan 13:S2213-2198(23)00050-8. doi: 10.1016/j.jaip.2022.12.039. Online ahead of print.ABSTRACTChronic granulomatous disease (CGD) is a rare inborn error of immunity, resulting from a defect in nicotinamide adenine dinucleotide phosphate (NADPH) oxidation and decreased production of phagocyte reactive oxygen species. The main clinical manifestations are recurrent infections and chronic inflammatory disorders. Current approaches to management include antimicrobial prophylaxis and control of inflammatory complications. Hematopoietic stem cell transplantation (HSCT) or gene therapy can provide...
Source: Cancer Control - January 16, 2023 Category: Cancer & Oncology Authors: Alexander H Yang Brigit Sullivan Christa S Zerbe Suk See De Ravin Andrew M Blakely Martha M Quezado Beatriz E Marciano Jamie Marko Alexander Ling David E Kleiner John I Gallin Harry L Malech Steven M Holland Theo Heller Source Type: research
Eleven science stories likely to make big news in 2023
As the COVID-19 pandemic enters its fourth year as a global health emergency, researchers will continue pushing to help make the disease manageable and ordinary. They will track hundreds of subvariants of Omicron, the highly transmissible but seemingly less lethal strain of SARSCoV-2 that dominated in 2022. Virologists will watch the virus’ evolution this year to see whether it has finally slowed or a more dangerous variant pops up, evading much of the immunity that humanity has built up to previous ones. Vaccine researchers hope to develop new shots that provide broad protection against a variety of coronaviruses. Ano...
Source: ScienceNOW - January 4, 2023 Category: Science Source Type: news
Early Win for Gene Therapy in Rare Form of SCID
(MedPage Today) -- A gene therapy for a rare type of severe combined immunodeficiency (SCID) was safe and sufficiently restored T-cell immunity in an early trial, results from a phase I/II study showed.
At a single center, all 10 infants with...
Source: MedPage Today Allergy - December 21, 2022 Category: Allergy & Immunology Source Type: news
