Italy, Genetics, Medicine
Network of experts join forces to fight rare disease Collaboration between research groups is key in tackling rare diseases such as auto-immune disease Myasthenia Gravis (MG). Indeed, the rarity of the disease means that it can be difficult to collect enough samples of blood and tissues to perform quality research.
Renewed hope for gene therapy in rare disease Between 30 and 40 million people in Europe suffer from rare diseases —many of them children. As most of these diseases have genetic origins, gene therapy is a major hope for their future cure .
Picking the right virus candidate for gene therapy Viruses often get bad press. Likened to Trojan horses they are often associated with disease. But, i t is precisely because of their infectious nature that they can potentially be used as gene vectors - which are vehicles loaded with good copies of malfunctioning genes - and delivered to cells.
New laser to watch DNA-proteins interactions Within the framework of the European ATLAS project, a team of researchers in Naples has created a LASER-based prototype that could revolutionize medicine and our knowledge of the human genome .
New therapy against rare gene defects European scientists set up new therapeutic approaches to tackle specific Lysosomal Storage Disorders: a new drug combination and enzyme replacement therapy against Pompe disease and gene therapy against MPS V I (Mucopolysaccharidosis VI).
Two approaches to treat Lysosomal Storage Diseases Lysosomes are membrane-bound organelles found in most animal cells. They are responsible for treating cellular waste. Genetic mutations in lysosomal enzymes lead to lysosome malfunction and waste accumulation.
Advances in Treating Huntington’s Disease Juliet Ross is suffering from Huntington’s Disease, a rare neurological disorder that affects one in 10.000 people. Due to a genetic defect, her body produces a toxic protein that damages neurons in her brain .
Huntington's disease: A treatment in sight? A treatment to directly fight Huntington’s disease could finally be in sight . Until now, therapies for this severely debilitating genetic disorder have only focused on alleviating physical and psychiatric symptoms.