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.
Disruptive science can have a significant impact outside of our own domains of research and into our personal lives. Keeping abreast of these developments can help prepare and inspire.
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.
Multinational biopharmaceutical company UCB has agreed to invest £1 billion in UK research and development (R&D), the government has announced in its latest Life Sciences Sector Deal. In the deal, £75 million will be invested into the development of new AI driven diagnostic tests while £50 million will be invested in digital pathology programmes and £37.5 million into regional digital innovation hubs.
Baboons can live for up to 195 days with hearts taken from pigs and genetically engineered to avoid extreme immune reactions, three times longer than previous attempts, according to a report published in Nature journal.
The World Health Organization is establishing an expert panel to set guidelines and standards on the ethical and safety issues of gene editing, the body has announced. This follows the recent revelation that a scientist in China claimed he had edited the genes of twin babies to make them HIV resistant.
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.
Twin girls in China have allegedly been born after having their embryonic genetic code modified using CRISPR. Chinese researcher He Jiankui, from the Southern University of Science and Technology, claims to have turned off a gene called CCR5 to offer total protection against HIV, as well as smallpox and cholera.
A DNA vaccine tested in mice reduces accumulation of both types of toxic proteins associated with Alzheimer’s disease, according to research that scientists say may pave the way to a clinical trial.
New research from the University of Pittsburgh implicates the so-called “longevity protein” Klotho, both as culprit and therapeutic target.