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Study suggests postnatal gene therapy could treat Pitt-Hopkins syndrome
Researchers have found that restoring lost gene activity prevents many disease signs in an animal model of Pitt-Hopkins syndrome.
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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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CRISPR-dCas9 gene editing reverses brain genetic reprogramming caused by adolescent binge drinking
A new study in animals has shown that gene editing may be a potential treatment for anxiety and alcohol use disorder in adults who were exposed to binge drinking in their adolescence.
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Retinal cell map could advance precise cell and gene therapies for retinal diseases
Researchers have identified distinct differences among the cells comprising a tissue in the retina, findings that could help develop precise therapies for retinal diseases.
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Gene therapy: A potential cure for growth-hormone resistant dwarfism
Researchers have shown that gene therapy using a single-dose injection of a virus carrying the ‘good’ gene could possibly be used to cure growth-hormone resistant dwarfism.
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Using CRISPR to improve lung cancer treatments
Scientists have developed CRISPR with a new process that evaluates the impacts of gene edits that alter rather than deleting DNA code.
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Researchers use brain organoids to identify neural abnormalities and test gene therapy tools
Scientists used human brain organoids to reveal how a genetic mutation associated with a profound form of autism disrupts neural development, and gene therapy tools to recover the gene’s function effectively rescued neural structure and function.
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Study shows muscular dystrophies can be repaired with CRISPR-Cas9
Researchers have introduced a CRISPR-Cas9 tool into human muscle stem cells using mRNA, thus discovering a suitable method to treat muscular dystrophies.
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New CRISPR gene-editing technology could lead to better treatments for HIV
Scientists at Northwestern Medicine have developed new techniques in human blood to pave potential paths towards a HIV cure.
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Novel genome-wide approach to engineer more effective immunotherapy
Scientists have developed a genetic screening platform to identify genes that can enhance immune cells to make them more persistent and increase their ability to eradicate tumour cells.
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Research collaboration to study ageing reversal in liver tissue established
Genflow have announced a scientific research collaboration with the University of Rochester’s Aging Research Center. Together, they will study the ageing process in liver tissue.
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New genetic model of Down’s syndrome developed in rats
Scientists have created what they believe is the first rat model of Down’s syndrome. The animal model shares features with human Down’s syndrome, which will allow for efficient testing of new therapies.
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Novel approach for discovering cancer drug targets
A next-generation spatial genomics technology has allowed scientists to link specific genes to complex tumour characteristics at a scale and resolution not previously possible.
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Vaccine to potentially protect against disfiguring skin disease developed
Using CRISPR gene-editing technology, scientists have developed a vaccine designed to prevent infection by Leishmania major.
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New study highlights the possibility to target gene editing in the lungs
Researchers have been able to significantly reduce tumours in a pre-clinical model of a rare genetic lung disease.
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Ncardia appoints Andy Holt as Chief Commercial Officer
An iPSC technology leader taps industry veteran to drive growth in cell therapy and drug discovery markets.