Data has shown the success of a new therapy to combat Leber congenital amaurosis (LCA), one of the leading causes of blindness in children, in LCA patients with CEP290 mutations. CEP290 acts as a barrier between two compartments of photoreceptor cells in the eye which convert light into signals, creating vision. Blindnesses caused by CEP290 […]
Researchers have announced that tetrahydrocannabinol (THC), an active ingredient in marijuana, affects epigenetics, triggering structural and regulatory changes in the DNA of users’ sperm.
Telbivudine, a drug currently in use for treating patients with hepatitis, could be of use in significantly slowing the progression of Amyotrophic lateral sclerosis (ALS), scientists from the University of Alberta have announced.
A new compound which blocks the most common genetic cause of familial ALS has been announced by scientists from the University of Alberta.
The latest annual report from Trinity Partners has found that orphan disease drugs often perform poorly at launch and continue to underachieve after three years of sales. At the other end of the spectrum, the most successful drugs on the market were for non-orphan diseases such as cancer and HIV.
A modified version of CRISPR has been used to reverse genetic obesity in two different mouse models without editing any genes. The technique uses the guidance system in CRISPR to target certain genetic sequences and amplifies existing gene activity to ramp up protein production.
The first findings from a comprehensive genomic analysis of the human brain have been unveiled, potentially uncovering a good deal about the inherited component of diseases such as schizophrenia and autism.
Eleven new genes associated with epilepsy have been discovered by an international team of scientists, advancing knowledge of the underlying causes of the condition and potentially helping in the development of new treatments.
Personalised tumour-detecting cells from adult skin cells have been used to shrink brain tumours in mice by up to 5%, scientists have revealed. While the strategy has not yet been fully tested in people, it could in the future give doctors the ability to develop a custom treatment for certain cancer types.
Amgen and Entera Bio are partnering up to develop new treatments for inflammatory disease and certain other serious illnesses using the Entera drug discovery program. The platform will be used to develop oral formulations for one preclinical large molecule program which Amgen has selected. Entera’s CEO said the collaboration would be an important validation test of the platform technology.
The CCR5 gene has been researched by scientists since the 1990s, and has a number of roles which have not yet properly been uncovered. Loss of the gene’s function is known, however, to increase the risk of potentially fatal reactions to some diseases, and has shown an ability to enhance learning in mice.
Scientists from the University of Pennsylvania have created a test using gene-editing tools such as CRISPR to identify a gene variant responsible for severe hypertrophic cardiomyopathy (SHC). SHC is an often-familial disease which thickens heart walls and is linked to a variant in the TNNT2 gene.
A new deal created by the Coalition for Epidemic Preparedness Innovations (CEPI) will use $8.4m to facilitate development of a vaccine platform to fight unknown pathogens. Under the deal, Imperial College London will work to create a self-amplifying RNA vaccine platform, which can then be made to rapidly develop anti-pathogen vaccines.
AstraZeneca and Cancer Research UK (CRUK) have announced that they will work together to open a new research centre in the UK, applying CRISPR and other functional genomics technologies to develop new cancer drugs. Specifically, the centre will study how genes and proteins interact with each other in cancer cells, and create disease models using genome-altering technologies based on this.
An innovative gene therapy treatment for Parkinson’s seems to relieve symptoms by rewriting the brain, scientists have found. Following the success of the therapy study, the researchers are planning a larger trial to start at the end of 2019.