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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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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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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.
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P53 could be key to therapies for salivary gland cancer
Mouse models show that activating a non-mutated form of the gene could lead to developing therapies for salivary gland cancer.
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CAR-T therapy becomes a powerful tool against an autoimmune disease like MS
US researchers have shown that CAR-T therapy can be used to eliminate unwanted cells that cause autoimmunity.
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Drug Target Review Cell and Gene Therapy Supplement 2022
This supplement focuses on how cell and gene therapy can target diseases such as cancer and reduce elevated lipoprotein(a).
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CIRM grant $4.8m to fund gene therapy for rare disease
CIRM grant will fund novel gene therapy that aims for single lifelong treatment of Friedreich’s ataxia, a progressive neuromuscular disorder; a second CIRM grant will advance efforts to leverage UC San Diego research on another rare disease
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New target in the fight against heart disease
US scientists have new insight into how heart cells enable unhealthy growth, and identify a new target to intervene against heart disease.