All Rare & Genetic Disorders articles – Page 5
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NewsCrohn’s disease: discovery of two distinct molecular subtypes
By studying organoids derived from adult stem cells, researchers made a discovery that could improve the development of personalised therapeutics.
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ArticleScaling genetic medicine with tRNA
Tune into this episode which explores how engineered tRNAs could treat thousands of rare diseases and cancers that share the same mutation.
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NewsDamaged small intestine repaired with hPSC-derived organoids
Cincinnati Children’s have repaired damaged intestines in a rodent model, moving research efforts closer to clinical trials.
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NewsmRNA vaccine provides enhanced protection against mpox
The mRNA vaccine candidate more effectively limited symptoms and disease duration in non-human primates.
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ArticleExploring the potential of ADCs beyond oncology
Antibody Drug Conjugates (ADCs) are already deployed as part of the roster of oncology treatments, but here, Mike Ward, Head of Thought Leadership for Life Sciences & Healthcare, Clarivate, looks into their potential to help treat a whole range of other diseases and conditions. He discusses the barriers and enablers ...
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NewsSpinal muscular atrophy: searching for a cure
Organoids with SMA-pathology uncovered key findings about the disease, which could be utilised to develop new therapeutic options.
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NewsPrime editing corrects the CFTR gene mutation
The approach precisely and durably corrects the CFTR mutation in human lung cells, which could lead to superior treatments.
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ArticleSmall molecules for regenerative medicine
In this episode, we discuss how new insights into stem cell biology are leading to novel therapeutics in regenerative medicine beyond cell therapy.
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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.