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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.
Despite the promise of gene therapies, significant challenges have emerged in the field. Dr Carsten Brunn discusses the current obstacles and opportunities when developing gene therapies.
A CRISPR gene editing technique has been developed to restore dystrophin, which is missing in many Duchenne muscular dystrophy (DMD) patients.
A new gene therapy that introduced SynCav1 to the brains of Alzheimer's mouse models was shown to preserve neuronal and synaptic plasticity.
Delivering Txnip to mice with retinitis pigmentosa was an effective treatment, making this approach a potential gene therapy for the disease.
Sahm Nasseri discusses promising pre-clinical results of an RNA-based therapeutic developed to treat retinitis pigmentosa type 11.
Researchers have shown that prime editing is able to effectively edit genes in mice but without the off-target effects of CRISPR.
Promising results have been shown in an animal model of Alzheimer's disease treated with zinc finger protein transcription factors.
Dr Ying Kai Chan discusses his latest research into how the delivery of AAV vectors for gene therapies can be made safer and more effective.
Researchers have reported that nano-micelles can be used to efficiently deliver CRISPR-Cas9 to edit genes in the brains of mice.
A new CRISPR gene therapy for chronic pain has been shown in mice to temporarily repress a gene involved in sensing pain.
A study has used artificial intelligence to reveal adeno-associated virus (AAV) capsid variants for use in gene therapies.
TALEN was shown to be almost five times more efficient than CRISPR-Cas9 at locating and editing genes in heterochromatin.
Treating only a few nerve cells with the hyper-interleukin-6 (hIL-6) gene therapy stimulated the regeneration of nerves.
A long-term study of macaques given mitochondrial replacement therapy (MRT) found that both treated individuals and their offspring were healthy and developed normally.
Professor Laurence D Hurst explains why understanding the nucleotide mutations in viruses, including SARS-CoV-2, can have significant implications for vaccine design.
