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Gene Therapy is the introduction of normal genes into cells in place of missing or defective ones in order to correct genetic disorders.
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.
The researchers developed nanoparticles able to penetrate the neural retina and deliver mRNA to the photoreceptor cells whose proper function makes vision possible.
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.
Discover how Catalent can help you achieve efficiencies and develop a robust, reproducible manufacturing plan for late-stage and commercial launch.
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.
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.
Japanese researchers proposed using RNA interference for correcting a mistake in the genetic code of patients with Fukuyama Muscular Dystrophy (FCMD).
US researchers have discovered a link between protein misfolding and liver cancer, that could help improve gene therapy for haemophilia.
Mouse models show that activating a non-mutated form of the gene could lead to developing therapies for salivary gland cancer.
US researchers have shown that CAR-T therapy can be used to eliminate unwanted cells that cause autoimmunity.
This supplement focuses on how cell and gene therapy can target diseases such as cancer and reduce elevated lipoprotein(a).
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
US scientists have new insight into how heart cells enable unhealthy growth, and identify a new target to intervene against heart disease.
A new Drug Target Review issue is now ready to download! This issue features articles which explore how artificial intelligence can enhance screening and ways to find new hits through simultaneous orthogonal screens. Also included are articles on CRISPR, immuno-oncology and RSV vaccines.
In primate models, researchers have successfully shown that developed AAVs can cross the blood-brain barrier, which keeps many drugs from getting into the brain.
