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.
Efficiently translating genomic research into the clinic is one of the most important steps in the development of the field. The clinic is where we will see things come to fruition.
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.
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.
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.
Time to pop the champagne – 100,000 genomes have been reached. Well done Genomics England!
A universal test which can detect traces of any cancer in a patient’s bloodstream in ten minutes has been developed by scientists from the University of Queensland. As it stands the test has a sensitivity of 90%, so is able to detect 90 in 100 cases of cancer.
Certain genetic processes of neurodegeneration, as seen in dementia, have been identified by a research team led by UCLA scientists. Two major groups of genes which create over-productions of the tau protein, which is integral to loss of neurons seen in major dementia forms, were found using mouse models of dementia, although the researchers performed additional experiments which determined the same processes occur in human brains.
There is a large genetic component to attention deficit/hyperactivity disorder (ADHD), a study published recently in Nature Genetics has confirmed. The study assessed 20,183 individuals diagnosed with ADHD and 35,191 controls, finding variants in 12 independent loci which all contribute to ADHD.