Researchers have used high-throughput screening on AAV vector capsid libraries to identify which ones are best for certain gene therapies.
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Gene Therapy is the introduction of normal genes into cells in place of missing or defective ones in order to correct genetic disorders.
Researchers have shown a gene therapy to correct the genetic mutation that causes Danon disease is successful in pre-clinical trials.
A study has shown that altering amino acid residues in Cas9 to produce new variants can produce a vector with increased gene editing specificity.
Researchers have developed a new regenerative gene therapy using neurogenic differentiation, which has shown efficacy treating Huntington's disease in mice.
Researchers have created a method to activate CAR T cells with blue LED light to successfully combat solid tumours in mice.
Results of an international collaboration show promise for the future of motor neurone disease treatment efforts, as protective gene is identified.
Researchers have developed a new CRISPR technique, using a minigene, which was inserted into mouse DNA, resulting in improved liver disease symptoms.
Mice with congenital blindness have shown significant improvement in vision after undergoing a new gene therapy.
A study has demonstrated that exosomes can be used as nanocarriers for therapeutic contents, as a new type of gene therapy.
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…
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…
A recent study has shown that transplanting new inhibitory neurons may repair damaged brain circuits.
New cell experiments show more effective genetic 'cuts' that could one day become the foundation of more effective gene therapies.