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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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Therapeutics
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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.
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Ground-breaking small molecule attenuates neuroinflammation in mice
Limiting neuroinflammation was shown to be a promising approach to treat neurological diseases such as stroke and spinal cord injury in mice.
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Sanofi acquires Translate Bio for mRNA therapeutic development
Sanofi has completed the acquisition of mRNA therapeutics company Translate Bio for a total of approximately $3.2 billion.
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Mechanism discovery provides new model for studying multiple sclerosis
The over-expression of a growth factor receptor resulted in hypomyelination, leading to diseases such as multiple sclerosis in mouse models.
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Exosome-based approach reduces levels of HIV in mice
Scientists have used exosomes to deliver a novel protein that prevented HIV from replicating in the bone marrow, spleen and brain of mouse models.
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Nasal drugs show promise for slowing Parkinson’s disease
Two different peptides were found to slow the spread of alpha-synuclein, potentially becoming the first drugs to slow the progression of Parkinson’s disease.
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Drug Target Review Synthetic Biology ebook 2021
In this ebook, discover why CRISPR can aid the development of new drugs via loss-of-function screens and how the field of synthetic biology is likely to evolve within the context of health.
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Club cell factors may reverse immune suppression in tumours
New research has revealed that club cells secret anti-immunosuppressive factors in tumours, potentially revolutionising cancer therapies.
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Eczema may stem from poorly regulated sex hormones, study finds
A breaking discovery has revealed the gene HSD3B1 that regulates the production of sex hormones is linked to atopic dermatitis (AD).