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Exosomes used by researchers as form of novel gene therapy
A study has demonstrated that exosomes can be used as nanocarriers for therapeutic contents, as a new type of gene therapy.
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Genomics
Genomics is the branch of molecular biology concerned with the structure, function, evolution, and mapping of genomes.
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Extensive tumour protein analysis reveals new therapy targets
New research has categorised hundreds of cancers based on their common protein mutations, highlighting cell components and tumour microenvironments as possible new therapy targets.
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CRISPR: kick-starting the revolution in drug discovery
Gene editing using the CRISPR system has been established as the most powerful tool in the search for new drugs and is now being exploited for therapeutic purposes. Here, Pushpanathan Muthuirulan discusses the promises and wider opportunities of using CRISPR technology to open up the possibility of large-scale screening of…
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Championing genomics in the UK
Professor Sir John Bell, Regius Professor of Medicine at the University of Oxford, delivered a speech at the MHRA’s 14th Annual Lecture in London, outlining his vision for the UK life sciences industry. Here, Nikki Withers summarises the key take-home messages from the talk, including how UK researchers and investors…
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Epigenome editing used to reverse gene mutation in mice
A study has demonstrated the success of changing the genome of mice, regulating the production of the C11orf46 gene.
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DNA successfully cut and inserted using Class 1 CRISPR system
A new study has shown that a Class 1 CRISPR gene editing system can achieve functional DNA repairs in human cells with no prominent off-target effects.
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Drug Target Review – Issue 4 2019
This issue includes a discussion on the future of high-throughput screening through collaboration, an analysis of mass spectrometry as a structural biology tool and an exploration of the challenges of hit-to-lead when researching tropical diseases. Also in the issue are articles on immuno-oncology and assays.
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New method to analyse CRISPR editing developed
Researchers have announced the creation of a novel technique for screening CRISPR edits that allows scientists to identify unintended outcomes in the genome.
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Fruit fly model to study mitochondrial diseases developed
Researchers have created ‘three-parent flies' which can be used as a model to study mitochondrial diseases and screen potential drug compounds.
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Researchers find mutation in CUL3 gene responsible for autism
Scientists have shown that the mutated CUL3 gene, which is involved in the cell’s clearance of proteins, plays a role in the development of autism and schizophrenia in mice.
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Mucin 6 gene indicates predisposition to late-onset Alzheimer’s
Scientists have idenified that variation in a gene called Mucin 6 appears to indicate a tendency for acquiring Alzheimer’s disease.
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ELRIG Drug Discovery 2019: looking back to the future
On 5-6 November, ACC Liverpool hosted the ELRIG Drug Discovery event, which allowed R&D professionals to come together and discuss the latest industry developments.
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Modified CRISPR gene editing tool could improve therapies
New cell experiments show more effective genetic 'cuts' that could one day become the foundation of more effective gene therapies.
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Gene therapy for epilepsy shows promise in animal models
An AAV-delivered gene therapy has demonstrated success in animal models of temporal lobe epilepsy, so could be a therapeutic option for human patients.
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Genetic mutation in monkeys identified as cause of Bardet-Biedl Syndrome
A study has revealed that in rhesus monkeys, a genetic mutation could be the cause of Bardet-Biedl Syndrome, providing a way to study the disease and develop therapies.