All Rare & Genetic Disorders articles – Page 5
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ArticleUncovering the genetic and cellular impacts of smoking
Epigenetic data assessing the effects of smoking on DNA methylation could serve as biomarkers of exposure effects.
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NewsExploring the role of class IIa HDACs in inflammatory diseases
The pharmacological inhibition of class IIa HDACs could be a therapeutic approach for addressing Th17-related inflammatory and autoimmune diseases.
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ArticleDeveloping an mRNA therapy for Duchenne muscular dystrophy
In this Q&A, Aki Ko, CEO and co-founder of Elixirgen Therapeutics, elucidates how their new mRNA technology could potentially restore muscle function in those suffering from Duchenne muscular dystrophy.
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NewsTruncated version of the SMN2 gene could revolutionise drug discovery
A condensed version of the SMN2 gene could improve discovery of potential therapies for spinal muscular atrophy and other conditions.
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NewsNew insights into viral-related myocarditis
Understanding heart changes at the molecular level during early infection with adenovirus could enable identification of those at higher risk of arrhythmia.
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NewsDOP-targeted therapies could exert significant anxiolytic effects
Researchers have uncovered the role of the PL-BLA neuronal axis and its potential function in DOP-mediated anxiolytic effects.
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NewsCombatting HIV using gene therapy
Researchers have created a nanomedicine loaded with siRNAs, which demonstrated a 73 percent reduction in HIV replication.
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CRISPR activation: quicker diagnosis of rare genetic diseases
Using CRISPRa to activate genes in readily accessible cells provides an effective and accurate diagnosis of genetic diseases.
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Blocking the IL-6 receptor to protect tissues from CRS
The new study’s findings suggest that CRS can be treated with an IL-6 receptor antibody that has a short half-life.
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NewsInvestigating neuron-OPC synapses in live tissue
Oligodendrocyte precursor cells and the synapses they form with neurons could be relevant to many disease conditions, including cancer.
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ArticleGrowth hormone’s influence on neuropsychiatric disorders
The discovery of a mechanism involving anxiolytic effects of GH may lead to the development of new classes of anxiolytic drugs.
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NewsModel for spinal muscular atrophy using human iPSCs
A 2D neuromuscular junction model enables high-throughput screening to discover new treatments for neuromuscular diseases.
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Novel AI algorithm improves decoding accuracy of brain signals
A new deep-learning method could enhance therapeutic devices for people with neurological or mental health conditions.
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AI system develops features of complex brains
An AI system could be used to observe how physical constraints shape brains and impact people with cognitive difficulties.
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NewsPotential biomarker found for inflammatory bowel disease
A new approach called reverse metabolomics has discovered molecules that may serve as a biomarker or a therapeutic target for IBD.
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ArticleDiscovering the principles of nervous system function
Dr William Schafer, of the MRC Laboratory of Molecular Biology, focuses on the cellular and molecular mechanisms of behaviour, which is one of the fundamental problems of biology. Schafer and his colleagues have studied the nematode Caenorhabditis elegans, using a variety of approaches such as optogenetic neuroimaging and high content ...
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NewsEngineered biofilm helps to tackle infections in CF patients
A living material resembling sputum from CF patients can grow biofilms, enabling scientists to assess the effectiveness of antimicrobials.
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NewsChemotherapy success calculated by new machine learning model
Percent necrosis calculated with machine learning model for patients with osteosarcoma provides an accurate prognosis for survival.
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NewsiPSC-derived cells can augment remuscularisation of the heart
Researchers have improved the method of regenerating heart muscle after a heart attack using a combination of iPSC-derived cells.
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ArticleProtein offers promise for treatment of reproductive conditions
Scientists found that CXCR4 protein expression outside of the uterus is important for pregnancy maintenance.