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New gene therapy could preserve vision in retinitis pigmentosa patients
Delivering Txnip to mice with retinitis pigmentosa was an effective treatment, making this approach a potential gene therapy for the disease.
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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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How RNA therapeutics can be used to treat retinitis pigmentosa
Sahm Nasseri discusses promising pre-clinical results of an RNA-based therapeutic developed to treat retinitis pigmentosa type 11.
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Prime editing demonstrates success at precise gene editing in mice
Researchers have shown that prime editing is able to effectively edit genes in mice but without the off-target effects of CRISPR.
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‘Zinc finger’ gene therapy could help to treat Alzheimer’s disease
Promising results have been shown in an animal model of Alzheimer's disease treated with zinc finger protein transcription factors.
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A strategy to cloak gene therapy AAV vectors from immune responses
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.
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Nano-micelles used in CRISPR genome editing of mouse brains
Researchers have reported that nano-micelles can be used to efficiently deliver CRISPR-Cas9 to edit genes in the brains of mice.
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CRISPR-dead Cas9 could offer non-addictive treatment for pain, study shows
A new CRISPR gene therapy for chronic pain has been shown in mice to temporarily repress a gene involved in sensing pain.
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Machine learning used to generate AAV capsids for gene therapy
A study has used artificial intelligence to reveal adeno-associated virus (AAV) capsid variants for use in gene therapies.
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TALEN is a more efficient gene editing tool for tightly packed DNA, finds study
TALEN was shown to be almost five times more efficient than CRISPR-Cas9 at locating and editing genes in heterochromatin.
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Hyper-interleukin-6 allows paralysed mice to walk again
Treating only a few nerve cells with the hyper-interleukin-6 (hIL-6) gene therapy stimulated the regeneration of nerves.
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Mitochondrial replacement therapy (MRT) shown to be safe in monkeys
A long-term study of macaques given mitochondrial replacement therapy (MRT) found that both treated individuals and their offspring were healthy and developed normally.
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Tweaking synonymous sites for gene therapy and vaccines
Professor Laurence D Hurst explains why understanding the nucleotide mutations in viruses, including SARS-CoV-2, can have significant implications for vaccine design.
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New gene therapy could treat dominant optic atrophy
Researchers have found that an OPA1-targeted gene therapy can treat dominant optic atrophy in pre-clinical trials.
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CRISPR: a molecular scissor set to change the world
In October this year, Emmanuelle Charpentier and Jennifer Doudna, the two scientists who pioneered the revolutionary gene-editing technology CRISPR, were awarded the Nobel Prize in Chemistry. Here, Pushpanathan Muthuirulan discusses the potential for this technology and the importance of using it safely, ethically and responsibly.
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‘Neuroregenerative’ study uses glial cells for stroke repair in primates
In rhesus macaques, a team were able to induce neural regeneration from brain internal glial cells, repairing damage from stroke.