Researchers aim to repurpose former experimental cancer therapy to treat muscular dystrophy

Duchenne muscular dystrophy is caused by a faulty gene that leads to progressive muscle weakness.
Source: National Institutes of Health (NIH) News Releases - Category: American Health Source Type: news

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Publication date: 11 December 2018Source: Cell Reports, Volume 25, Issue 11Author(s): John K. Lee, Darko Bosnakovski, Erik A. Toso, Tracy Dinh, Surajit Banerjee, Thomas E. Bohl, Ke Shi, Kayo Orellana, Michael Kyba, Hideki AiharaSummaryDouble homeobox (DUX) transcription factors are unique to eutherian mammals. DUX4 regulates expression of repetitive elements during early embryogenesis, but misexpression of DUX4 causes facioscapulohumeral muscular dystrophy (FSHD) and translocations overexpressing the DUX4 double homeodomain cause B cell leukemia. Here, we report the crystal structure of the tandem homeodomains of DUX4 boun...
Source: Cell Reports - Category: Cytology Source Type: research
(Rice University) Rice University bioengineers have combined a virus that infects moths with magnetic nanoparticles to create a potential new therapy for inherited genetic diseases like muscular dystrophy, sickle cell, cystic fibrosis, spinal muscular atrophy and some forms of cancer.
Source: EurekAlert! - Cancer - Category: Cancer & Oncology Source Type: news
Nearly all diseases have a genomic basis. This varies from rare ‘single-gene disorders’ such as Duchenne muscular dystrophy, to ‘complex’ or ‘polygenic’ diseases such as type II diabetes mellitus and ulcerative colitis. Cancer is a genomic disease in which tumour evolution is driven by serial acquisition of somatic mutations. Investigation of genetic disorders has been predicated on available technologies, which until recently have been expensive, slow, low-throughput and therefore limited in availability.
Source: Medicine - Category: Internal Medicine Authors: Tags: Medicine and other specialties Source Type: research
Our knowledge of the effect of metformin on human health is increasing. In addition to its ability to improve the control of hyperglycaemia, metformin has been shown to reduce the burden o,f ageing via effects on damaged DNA and the process of apoptosis. Studies have shown that metformin may reduce the risk of cardiovascular disease through influences on body weight, blood pressure, cholesterol levels and the progression of atherosclerosis. Studies also suggest that metformin may be beneficial for neuro ‐psychiatric disorders, cognitive impairment and in reducing the risk of dementia, erectile dysfunction and Duchenne mu...
Source: British Journal of Clinical Pharmacology - Category: Drugs & Pharmacology Authors: Tags: REVIEW Source Type: research
Lab Chip, 2018, Accepted Manuscript DOI: 10.1039/C8LC00553B, Critical ReviewGeorge Alexander Truskey A number of major disease states involve skeletal muscle, including type 2 diabetes, muscular dystrophy, sarcopenia and cachexia arising from cancer or heart disease. Animals do not accurately represent many... The content of this RSS Feed (c) The Royal Society of Chemistry
Source: RSC - Lab Chip latest articles - Category: Chemistry Authors: Source Type: research
Fight Aging! provides a weekly digest of news and commentary for thousands of subscribers interested in the latest longevity science: progress towards the medical control of aging in order to prevent age-related frailty, suffering, and disease, as well as improvements in the present understanding of what works and what doesn't work when it comes to extending healthy life. Expect to see summaries of recent advances in medical research, news from the scientific community, advocacy and fundraising initiatives to help speed work on the repair and reversal of aging, links to online resources, and much more. This content is...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
AbstractProgressive meningiomas that have failed surgery and radiation have a poor prognosis and no standard therapy. While meningiomas are more common in females overall, progressive meningiomas are enriched in males. We performed a comprehensive molecular characterization of 169 meningiomas from 53 patients with progressive/high-grade tumors, including matched primary and recurrent samples. Exome sequencing in an initial cohort (n = 24) detected frequent alterations in genes residing on the X chromosome, with somatic intragenic deletions of the dystrophin-encoding and muscular dystrophy-associatedDMD gene a...
Source: Acta Neuropathologica - Category: Neurology Source Type: research
(University of Illinois at Urbana-Champaign) In a new study in cells, University of Illinois researchers have adapted CRISPR gene-editing technology to cause the cell's internal machinery to skip over a small portion of a gene when transcribing it into a template for protein building. Such targeted editing could one day be useful for treating genetic diseases caused by mutations in the genome, such as Duchenne's muscular dystrophy, Huntington's disease or some cancers.
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news
Bolstering the notion that RNA should be considered an important drug-discovery target, scientists at Scripps Research have found that several existing, FDA-approved anti-cancer drugs may work, in part, by binding tightly to RNA, the regulators of the basic activities of life within cells. The research offers another approach for tackling diseases that have been considered "undruggable," including amyotrophic lateral sclerosis (ALS), muscular dystrophy, cystic fibrosis and certain cancers.
Source: World Pharma News - Category: Pharmaceuticals Tags: Featured Research Research and Development Source Type: news
(Scripps Research Institute) The research offers another approach for tackling diseases that have been considered 'undruggable,' including amyotrophic lateral sclerosis (ALS), muscular dystrophy, cystic fibrosis and certain cancers.
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news
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