All Rare & Genetic Disorders articles – Page 4
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NewsNew data shows benefits of automated blood exchange for sickle cell disease
New studies highlight the potential of automated blood exchange to improve outcomes for sickle cell disease patients.
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NewsTessera's gene editing offers hope for sickle cell patients
Find out how Tessera's latest advancements in gene editing bring promising solutions to treat sickle cell disease.
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ArticleOncology advances through the lens of women in STEM
Dr Pooja Hingorani opens up about her journey in oncology research, the obstacles she’s overcome as a woman in STEM, and her mission to transform cancer care.
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ArticleNew insights into the role of viral capsids in gene therapy safety
Viral capsid assembly and quality are key factors in gene therapy success and safety. Here, Dr Chelsea Pratt explores how cutting-edge tools are enabling scientists to tackle these challenges and improve patient outcomes.
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NewsBreakthrough trial offers new hope for cystic fibrosis patients
A promising new inhaled therapy is progressing in Phase 2 trials, presenting new opportunities for improving cystic fibrosis treatment.
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NewsAI for drug discovery in advanced pain management
A deep-learning model found metabolites and drugs that could be repurposed for non-addictive and non-opioid options to treat chronic pain.
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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.