All Rare & Genetic Disorders articles – Page 5
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ArticleThe evolution of cell therapy to address unmet medical needs
We had the privilege of interviewing Brian Culley, CEO of Lineage Cell Therapeutics. He discusses how Lineage’s pluripotent stem cell platform is extraordinarily advantageous, details two early-stage programmes addressing auditory and vision disorders and reveals how Lineage is choosing to target conditions that will make a real difference.
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NewsNew ENaC blocker demonstrates an extended duration of action
The novel drug, ETD001, could provide an improved approach for mucus clearance in cystic fibrosis patients.
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NewsEpigenetic dysregulation mechanism in Crohn’s disease discovered
Intestinal epithelial organoids highlight a pathway implicated in CD, named major histocompatibility complex (MHC)-I.
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NewsNew biological pathway in IBD discovered
Researchers have identified a new biological pathway driving IBD and similar conditions that could be targeted with MEK inhibitors.
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NewsNew first trimester screening algorithm for preeclampsia
The screening model combines maternal history, ultrasound data and several tests for blood markers to enable personalised treatment.
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NewsXCL1 could mitigate psychiatric condition risk in offspring
Adult anxious behaviour in offspring may be related to the early life proinflammatory state caused by the absence of elevated XCL1.
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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.