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Gene found to protect motor neurons against degeneration
Results of an international collaboration show promise for the future of motor neurone disease treatment efforts, as protective gene is identified.
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Gene Therapy
Gene Therapy is the introduction of normal genes into cells in place of missing or defective ones in order to correct genetic disorders.
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CRISPR ‘minigene’ used to prevent genetic liver disease in mice
Researchers have developed a new CRISPR technique, using a minigene, which was inserted into mouse DNA, resulting in improved liver disease symptoms.
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Gene editing platform improves vision in blind mice
Mice with congenital blindness have shown significant improvement in vision after undergoing a new gene therapy.
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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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Using droplet digital PCR to monitor the safety of CAR T cells
CAR T-cell therapy has caused quite a stir in the immunotherapy world, but it is a process that requires precision and care. Quantitative polymerase chain reaction (qPCR) has thus far been the stalwart technique for providing a check on copy numbers, but here Ping Jin explains why droplet digital PCR…
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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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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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New cell therapy improves memory and stops seizures after brain injury
A recent study has shown that transplanting new inhibitory neurons may repair damaged brain circuits.
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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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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.
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Researchers find DNA therapy could treat patients with DMD
A study has revealed that using DNA-like molecules to repair gene mutations in models could act as a successful therapy for patients.
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CRISPR used to treat Duchenne muscular dystrophy in mice
A study has shown that CRISPR can be used as a regenerative technique to treat Duchenne muscular dystrophy, which could be developed as a therapeutic option for humans.