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New drug target identified for myelodysplastic syndrome
Researchers have uncovered how mutations affecting RNA splicing alter cells to develop myelodysplastic syndrome (MDS).
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Therapeutics
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Small molecule RGX-202 prevents metastasis in colorectal cancer
RGX-202 was found to foil a key pathway that cancer cells rely on for energy in mice, presenting a possible new colorectal cancer therapy.
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Novel “living medicine” could treat antibiotic-resistant infections
Researchers have created the first “living medicine” to treat antibiotic-resistant bacteria growing on the surfaces of medical implants.
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UC Riverside granted $5 million for stem cell research training
A $5 million grant will be used to train students in stem cell research to contribute towards new stem cell-based therapeutics.
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Two-week-old human embryos reveal origin of Huntington’s disease
Scientists have discovered a signalling pathway alteration in embryos with Huntington’s disease, paving the way for ground-breaking treatments.
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Psilera to collaborate with NIDA on pre-clinical psychedelic studies
Psilera will participate in two studies with the National Institute on Drug Abuse to combat addiction using a novel DMT formulation.
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Gene therapy method could restore visual function after stroke
Scientists have revealed a way to use gene therapy to turn glial brain cells into neurons, restoring vision and potentially restoring motor function.
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Utilising aquaporins could be the key to repairing corneal defects
New evidence suggests that aquaporin 5 (Aqp5) induction promotes corneal epithelial regeneration, which could lead to possible therapies.
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Zaki syndrome: the newly uncovered condition and its potential cure
Dubbed 'Zaki syndrome', the condition affects prenatal development of several organs and was identified using whole genome sequencing.
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Discussing next-gen AAV vectors with Dr Arun Srivastava, University of Florida
An interview with Dr Arun Srivastava about his research into safe and effective AAV vectors that do not prompt a reaction from the immune system.
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CRISPR screening tool exposes new leukaemia drug target
A CRISPR screening tool identified a new target for acute myeloid leukaemia with fewer side effects than current approaches.
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NINDS awards Sinopia $3.3m for development of Parkinson’s drug candidate
Sinopia has been awarded a $3.3 million Fast-Track SBIR grant to fund the study for its small molecule candidate for Parkinson’s disease.
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“Ultrasensitive” HPV detection paves way for precision treatment
A new study sequenced tumour DNA for the “ultrasensitive” detection of HPV, further honing precision treatment of the illness.
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Promising pre-clinical data announced for Parkinson’s drug
Stealth BioTherapeutics reported positive data from a pre-clinical study evaluating SBT-272 in a murine model of Parkinson's disease.
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New AI technology could shape the future of RNA therapeutics
Artificial intelligence was shown to predict the 3D shapes of RNA molecules, which could significantly advance RNA therapeutics.
