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No-cut CRISPR could be used to treat muscular dystrophy
Researchers have successfully used a no-cut CRISPR gene editing technique as a therapy for muscular dystrophy in mice models.
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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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New system enables improved pre-clinical testing of heart drugs
Researchers have extended the life of heart segments from 24 hours to six days, enabling pre-clinical trials to experiment on these new models for longer periods of time.
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Novel nanoparticles improve CRISPR gene editing
A study has used new synthetic lipids to deliver CRISPR gene editing tools into cells with up to 90 percent efficiency.
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CRISPR-Cas9 gene editing imaged for first time
Researchers have generated images of CRISPR-Cas9 gene editing for the first time, enabling improvement of the technique.
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New discovery shows ability of Schwann cells to generate myelin
Research from Oregon Health & Science University could lead to new therapies to heal nervous system disorders.
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Researchers develop directed evolution technique
New method for directed evolution could improve gene therapies and generic drug development.
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Potential cure for HIV revealed through combined therapies
A new study has used combined therapies to eliminate HIV from mice models, providing potential future cures.
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Source of nucleic-acid asymmetry could improve gene therapies
Researchers have analysed the physical origin and biological consequences of DNA-RNA hybrids which could inform gene therapies.
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Gene discovery could make cancer therapies more effective
A recent study has identified the DBC1 gene which leaves cancer cells more exposed to therapies.
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Longest successful gene expression raises hopes for gene editing therapies
A study has reported that genes delivered to rhesus monkeys are still being expressed four years later.
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Biomarkers for potential cancer immunotherapy identified
Researchers have found two biomarkers which play a significant role in the invasion of tumours by T-cells.
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MS treatment potential after condition-causing genes identified
Researchers have found 12 genes which cause MS, creating potential for preventative treatment of the condition.
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EU conditional marketing authorisation announced for ZYNTEGLO
ZYNTEGLO™ is the first gene therapy approved for transfusion-dependent β-thalassemia (TDT).
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Genetic therapy can heal damage caused by a heart attack
Researchers from King's College London have found a method that can induce heart cells to regenerate after a heart attack.
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Colon cancer proteins and gene analysis uncovers potential new treatments
A new study analysing both the entire set of genes and all the proteins produced by colon cancer tissues has revealed a more comprehensive view of the tumour.
