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Gene therapy could cure Danon disease, life-threatening genetic condition
Researchers have shown a gene therapy to correct the genetic mutation that causes Danon disease is successful in pre-clinical trials.
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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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TAZ gene therapy reverses Barth syndrome in mice
Researchers have successfully treated cardiac dysfuntion in mice models of Barth syndrome by using a gene therapy to replace TAZ.
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Researchers create new Cas9 variant to reduce off-target CRISPR edits
A study has shown that altering amino acid residues in Cas9 to produce new variants can produce a vector with increased gene editing specificity.
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New gene therapy for Huntington’s demonstrates success in mice
Researchers have developed a new regenerative gene therapy using neurogenic differentiation, which has shown efficacy treating Huntington's disease in mice.
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Using blue light to control CAR T cells and destroy tumours
Researchers have created a method to activate CAR T cells with blue LED light to successfully combat solid tumours in mice.
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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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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.
