news
Gene therapy for Morquio A disease successful in rat model
A novel gene therapy has fully corrected whole-body alterations in a rat model, paving the way for Morquio A therapies.
List view / Grid view
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.
article
Delivering gene therapies to the brain to treat Alzheimer’s
Dr Ronald G Crystal, Professor and Chairman of the Department of Genetic Medicine, Weill Cornell Medical College, spoke to Drug Target Review’s Fraser Owen about his research into Alzheimer’s disease and why gene therapies represent a promising area of research for neurodegenerative conditions.
news
Brain organoids mimic autism-related head size changes
Scientists have developed brain organoids that recapitulate the head size of autism patients to study the condition as well as possible therapies.
news
Cartilage growth gene TRPV4 shows promise for joint repair therapy
The discovery that the TRPV4 gene regulates cartilage growth could lead to treatments for osteoarthritis and other cartilage diseases.
video
Drug Target Review update: exclusive report, online summit and more!
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!
news
New skin cell mechanism discovery could treat muscle-related diseases
Scientists have identified the mechanism behind the conversion of skin cells into immature muscle cells, which could lead to therapies preventing muscle degeneration.
news
TREM2 regulation to prevent neurodegeneration is possible, study shows
Scientists used a synthetic thyroid hormone in mice to regulate the TREM2 gene implicated in diseases such as Alzheimer’s and Parkinson’s.
news
Prime editing method corrects cystic fibrosis in stem cells
A team of scientists used a CRISPR-Cas9 technique known as prime editing to correct cystic fibrosis in cultured human stem cells.
news
CRISPR-Cas9 used to prevent Fuchs’ corneal dystrophy in mice
Scientists have shown that start codon disruption with CRISPR-Cas9 gene editing can prevent Fuchs’ corneal dystrophy in mouse models.
news
Mitochondria repair in mice may provide answers on Parkinson’s disease
Researchers used an experimental small molecule that helped restore the removal of damaged mitochondria from brain cells in a mouse model of Parkinson's.
news
Genetic treatment reverses age-related memory loss in mice
Scientists have shown that manipulating the perineuronal nets (PNNs) in the brains of mice led to the reversal of age-related memory loss.
news
Prenatal editing halts progression of Hurler syndrome in pre-clinical model
US researchers used an AAV9 vector to edit a single base mutation in a prenatal mouse model, halting progression of Hurler syndrome.
article
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.
news
Novel gene therapy for heart failure moves closer to clinic
New research by US institutions in pigs shows that knocking down a particular signalling pathway after heart failure renews heart tissue.
article
Novel vectors for more efficient and less risky ocular gene therapy
Inherited blindness is the leading cause of vision loss in the working populations. Recently, the first gene therapy for the treatment of retinal dystrophy caused by mutations in the RPE65 gene became available. This gene therapy is based on adeno-associated virus (AAV) vectors injected under the retina. Subretinal injection is…
