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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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
Johnson &Johnson (NYSE:JNJ) opened its latest life science incubator in New York City, the healthcare giant said today. The 30,000-square-foot JLabs @ NYC is a collaboration between Johnson &Johnson Innovation, New York State and the New York Genome Center. Sited at the genome center in SoHo, the incubator is home to 26 startups and has room for four more, New Brunswick, N.J.-based J&J said. “Johnson &Johnson has deep entrepreneurial roots in New York and we are pleased to see our unique JLabs model applied in this rich ecosystem to foster the creation of new healthcare innovations that have the pote...
Source: Mass Device - Category: Medical Devices Authors: Tags: Funding Roundup Research & Development johnsonandjohnson Source Type: news
by Jing Zhao, Lei Zhang, Xiaodong Mu, Christelle Doebelin, William Nguyen, Callen Wallace, Daniel P. Reay, Sara J. McGowan, Lana Corbo, Paula R. Clemens, Gabriela Mustata Wilson, Simon C. Watkins, Laura A. Solt, Michael D. Cameron, Johnny Huard, Laura J. Niedernhofer, Theodore M. Kamenecka, Paul D. Robbins Nuclear factor κB (NF-κB) is a transcription factor important for regulating innate and adaptive immunity, cellular proliferation, apoptosis, and senescence. Dysregulation of NF-κB and its upstream regulator IκB kinase (IKK) contributes to the pathogenesis of multiple inflammatory and degenera ti...
Source: PLoS Biology: Archived Table of Contents - Category: Biology Authors: Source Type: research
In conclusion, in the Framingham Heart Study population, in the last 30 years, disease duration in persons with dementia has decreased. However, age-adjusted mortality risk has slightly decreased after 1977-1983. Consequences of such trends on dementia prevalence should be investigated. Recent Research on the Benefits of Exercise in Later Life A sizable body of work points to the ability of older individuals to continue to obtain benefits through regular physical activity, and particularly in the case ...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
Ichor Therapeutics is the most mature of the US-based companies that have emerged from the SENS rejuvenation research community in recent years. You might recall a number of interviews back in the Fight Aging! Archives with founder and CEO Kelsey Moody. He has his own take on how our community should proceed from laboratory to clinic: he is very much in favor of demonstrating (a) that the formal regulatory path offered by the FDA can work for the treatment of aging, and (b) that - given the right strategic approach - rejuvenation therapies can attract the attention, collaboration, and backing of Big Pharma entities in the ...
Source: Fight Aging! - Category: Research Authors: Tags: Healthy Life Extension Community Source Type: blogs
Abstract Like the major theme of a Mozart concerto, the immense and pervasive extracellular matrix drives each movement and ultimately closes the symphony, embracing a unique role as the fundamental mediator for most, if not all, ensuing intracellular events. As such, it comes as no surprise that the mechanism of just about every known disease can be traced back to some part of the matrix, typically in the form of an abnormal amount or activity level of a particular matrix component. These defects considerably affect downstream signaling axes leading to overt cellular dysfunction, organ failure, and death. From sk...
Source: Matrix Biology - Category: Molecular Biology Authors: Tags: Matrix Biol Source Type: research
Discussion There were no significant differences in diet consumption or in mouse weight gain between the control and experimental diets over the course of the 12 weeks of treatment, nor were there any observable adverse effects on the mice. Comparison of wire hang times over the 12 weeks of treatment using the maximum holding impulse (weight x time) 13 relative to the start of treatment (100%) as a measure revealed a slight decline in wire hang times in animals on the control diet (Figure 1). Isoflavones and isoflavones with BBI showed a slight and sustained increase in wire hang times over the course of the treatment, but...
Source: PLOS Currents Muscular Dystrophy - Category: Neurology Authors: Source Type: research
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