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Myasthenia Gravis is a rare autoimmune disease which leads to a fatigability of various muscles. Scientists know about the progress and symptoms, but not the actual cause of this disease. Now fourteen pairs of identical twins joined the EU funded medical project “Fight-MG”, to help find the cause and for a better treatment of the patients.
Support from a network of leading researchers across Europe specialised in a rare auto-immune disease with unmet medical needs could help test several novel treatments
Scientists within the European research project AIPgene have developed a new gene therapy for Acute Intermittent Porphyria (AIP).
New virus serotypes are safely used as ‘DNA transporters’ to successfully deliver genes to deficient cells.
An early stage clinical trial on patients with the rare metabolic disorder, Pophyria, has shown no side effects—further trials, if successful, could lead to the first gene therapy of its kind.
Metabolites found in our blood are linked to ageing and can signpost the risk of developing age-related diseases. This may help avoid such risks and reduce the rate at which we age biologically.
Combining research on twins with new technology shows both nature and nurture play a role in biological ageing.
A promising new approach to gene therapy for neurodegenerative diseases makes use of an unexpected virus as the agent of delivery.
First tests of new approach based on gene therapy could improve treatments of diseases affecting the brain
Intervertebral disc degeneration can result in back pain. Now a team of scientists is debunking the myth that excessive physical work is the main culprit, and is looking at other causes.
Lower back pain is poorly understood. But genetic factors rather than spinal disc degeneration may provide a more robust understanding of its causes.
The human DNA has been decoded already in the Human Genome Project. Now scientists from Martinsried near Munich are taking the next step to decode all proteins of a human being.
How many types of proteins are there in a cell? How many of each type are there and where are they at any given time? New methods may shed light on these fundamental biological questions.
New insights into how large protein complexes function differently in healthy and sick cells has now been granted thanks to new technologies.
Protein-DNA binding is fundamental to gene regulation. Now, new methods based on laser pulses allow fast interactions between proteins and DNA to be followed.
The pattern of brain alterations may be similar in several different neurodegenerative diseases, which opens the door to alternative therapeutic strategies to tackle these diseases
Parkinson’s disease modifies a crucial circuit of the central nervous system in a specific way. This could open up an alternative therapeutic approach that avoids side effects of current therapies.
On 15th April is the 1st International Pompe Disease Day, a campaign to raise awareness of this rare but severe gene defect. Pompe Disease is only one of more than 40 metabolic disorders that mainly affect children under the age of 10, often with devastating consequences. Now scientists of the European research project EUCLYD setting up new therapeutic methods to tackle these gene defects.
Enzyme therapy proves effective in treating LSDs, whilst gene therapy is an upcoming contender.