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DTR’s news round-up 2019: the stories that defined the year
Drug Target Review lists its 10 most popular news stories from 2019, summarising the drug targets that you wanted to read about.
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Genome Editing
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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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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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Study shows that CRISPR-Cas9 gene editing has a low risk of mistakes
A study has shown that unintended mutations from gene editing with CRISPR-Cas9 are rare in zebrafish, providing reassurance that this technology is a valid tool with great promise for the treatment of genetic disorders.
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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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New approach to treating incurable leukaemia in children discovered
A new study into the molecular causes of acute lymphoblastic leukaemia could lead to a new target for therapy.
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How will CRISPR evolve in the future?
CRISPR is a tool used by researchers to precisely edit genes and has shown potential for treating genetic diseases. This article delves into some recent developments and explores what the future holds for CRISPR.
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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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New gene editing tool could correct 89 percent of genetic defects
'Prime editing', a new CRISPR genome-editing approach, is capable of directly editing human cells in an accurate and efficient way.
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Full gene-replacement model developed for Alzheimer’s MAPT gene research
Researchers in the US have successfully produced a mouse model with a human MAPT gene to enable more accurate research into Alzheimer’s therapy.
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Gene therapy for DMD safely preserves muscle function
Gene therapy for the treatment of Duchenne muscular dystrophy has safely stopped the muscle deterioration associated with the disease.
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Researchers use new CRISPR method to alter specific bacteria strains
A study has demonstrated how to use CRISPR to deliver DNA to particular bacteria, which could be used as an alternative to antibiotics.
