List view / Grid view
Gene Therapy is the introduction of normal genes into cells in place of missing or defective ones in order to correct genetic disorders.
A novel gene therapy has fully corrected whole-body alterations in a rat model, paving the way for Morquio A therapies.
Dr Ronald G Crystal, Professor and Chairman of the Department of Genetic Medicine, Weill Cornell Medical College, spoke to Drug Target Review’s Victoria Rees about his research into Alzheimer’s disease and why gene therapies represent a promising area of research for neurodegenerative conditions.
Scientists have developed brain organoids that recapitulate the head size of autism patients to study the condition as well as possible therapies.
The discovery that the TRPV4 gene regulates cartilage growth could lead to treatments for osteoarthritis and other cartilage diseases.
Hear about the latest Drug Target Review updates from Deputy Editor Victoria Rees who discusses our new report on AI & Informatics, the Cell & Gene Therapy Advancements online summit and much more!
Scientists have identified the mechanism behind the conversion of skin cells into immature muscle cells, which could lead to therapies preventing muscle degeneration.
Scientists used a synthetic thyroid hormone in mice to regulate the TREM2 gene implicated in diseases such as Alzheimer’s and Parkinson’s.
A team of scientists used a CRISPR-Cas9 technique known as prime editing to correct cystic fibrosis in cultured human stem cells.
Scientists have shown that start codon disruption with CRISPR-Cas9 gene editing can prevent Fuchs’ corneal dystrophy in mouse models.
Researchers used an experimental small molecule that helped restore the removal of damaged mitochondria from brain cells in a mouse model of Parkinson's.
Scientists have shown that manipulating the perineuronal nets (PNNs) in the brains of mice led to the reversal of age-related memory loss.
US researchers used an AAV9 vector to edit a single base mutation in a prenatal mouse model, halting progression of Hurler syndrome.
How engineered RNA binding proteins delivered in vivo by gene therapy could treat myotonic dystrophy
In this article, Dr Jim Burns discusses promising pre-clinical results of how a new platform could treat the root cause of many devastating genetic diseases including myotonic dystrophy type 1.
New research by US institutions in pigs shows that knocking down a particular signalling pathway after heart failure renews heart tissue.