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Inflammation ‘brake’ gene may impact kidney disease outcomes
Australian researchers uncover a gene that controls inflammation in kidney disease, which could pave the way for more precise disease diagnostics and personalised treatments.
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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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Recombining drugs helps restore gene’s ability to signal for death of leukaemia cells
US researchers find combination of chloroquine and venetoclax promotes cancer cell death in mouse models with acute myeloid leukaemia (AML).
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Combination therapy prevails against BET inhibitor resistance
St. Jude Children's Research Hospital, US, scientists created a new combination therapy method to tackle drug resistance in a type of leukaemia with KMT2A gene rearrangement.
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Step forward in gene therapy to treat cause of sudden cardiac arrest
Using gene therapy, the researchers corrected abnormal heart rhythms by restoring a protein that heart cells need to establish connections with one another.
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Research shows lipid nanoparticles are highly effective in gene therapy
Lipid nanoparticles have been used to encapsulate CRISPR-Cas9 and deliver it to cells in mice, where it was highly effective at knocking down expression of a target protein.
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Usher syndrome: first nonhuman primate model
US researchers evaluate an experimental gene therapy in the first ever nonhuman primate model for Usher Syndrome.
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Therapeutic strategy using p53 induces cancer cell death
US researchers spotlight how p53, the most frequently mutated tumour suppressor gene, can be activated against cancer cells.
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Researchers develop novel method to insert large DNA sequences more accurately in cells
The scientists say that the engineered CRISPR enzymes could overcome key limitations for eventual use to treat genetic diseases irrespective of a patient’s particular mutation.
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New study highlights lipid nanoparticles and mRNA could treat blindness
The researchers developed nanoparticles able to penetrate the neural retina and deliver mRNA to the photoreceptor cells whose proper function makes vision possible.
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A change in genetic code causes lung cancer to develop
Using lung basal cell organoids, researchers identify a gene that directs the development of lung cancer and offers a better understanding of its disease treatment.
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Executive summary: Scale-up strategies for late-stage cell therapies
Discover how Catalent can help you achieve efficiencies and develop a robust, reproducible manufacturing plan for late-stage and commercial launch.
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Five recent cell and gene therapy discoveries
This article highlights five of the latest findings using cell and gene therapy techniques that could be used in the development or design of new therapies.
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Drug Target Review’s top content picks from 2022
In this article, Drug Target Review’s Izzy Wood and Ria Kakkad share some of the most ground-breaking moments from drug discovery this year.
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Novel gene therapy offers potential treatment for FCMD
Japanese researchers proposed using RNA interference for correcting a mistake in the genetic code of patients with Fukuyama Muscular Dystrophy (FCMD).
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Misfolding proteins bring caution for gene therapies for haemophilia
US researchers have discovered a link between protein misfolding and liver cancer, that could help improve gene therapy for haemophilia.