New cell experiments show more effective genetic 'cuts' that could one day become the foundation of more effective gene therapies.
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An AAV-delivered gene therapy has demonstrated success in animal models of temporal lobe epilepsy, so could be a therapeutic option for human patients.
'Prime editing', a new CRISPR genome-editing approach, is capable of directly editing human cells in an accurate and efficient way.
Researchers in the US have successfully produced a mouse model with a human MAPT gene to enable more accurate research into Alzheimer’s therapy.
Gene therapy for the treatment of Duchenne muscular dystrophy has safely stopped the muscle deterioration associated with the disease.
A study has demonstrated how to use CRISPR to deliver DNA to particular bacteria, which could be used as an alternative to antibiotics.
A study has revealed that a mutation in the NKAP gene causes a rare syndrome, providing researchers with the potential to develop treatments for the condition.
The newly identified variant could play a role in gene therapies that require high accuracy and precision.
A method designed to genetically alter laboratory mice can also be used to produce personalised animal models of an aggressive type of malignant brain cancer in children.
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.
A new study has shown a new mechanism of controlling fat production in the body which could lead to new therapies to treat obesity.
CRISPR is the buzzword of the moment in the drug discovery industry – mainly due to its potential to correct disease-causing mutations. However, those using the technology need to be mindful that it is used responsibly, and possible risks are considered before use. Mark Behlke discusses the potential of CRISPR…
Researchers have discovered a particular protein that is required for enterovirus replication and survival, presenting a therapeutic target.
A team has used a lentiviral capsid-based bionanoparticle system to deliver CRISPR-Cas9 gene editing therapies, reducing undesired effects.