Experimental treatment for Duchenne muscular dystrophy started in December 2017 under NCT03368742 assignation. Clinicians attempted to insert adeno-associated virus (AAV9),…
Gene Therapies
medically-justified modifications of people
In a study done by famous George Church’s lab, scientists demonstrated the use of genetic engineering in disease prevention. They…
Gaucher disease is caused by mutations in GBA gene, which deactivate beta-glucocerebrosidase enzyme. Patients suffer from accumulation of molecules normally…
Pyruvate kinase deficiency (PKD) is a rare genetic disorder, present in 5 / 100,000 people, caused by mutations in PKLR…
Tay-Sachs disease is caused by mutations in HEXA gene. Patients experience rapid neurodegeneration, including loss of neurons and decrease in…
Clinical trial (NCT04133649) just began recruitment stage. The procedure will consist of a single intravenous injection, followed by six safety…
Parkinson’s disease is one of the most frequent disorders of brain. Affected individuals experience impairment of muscle control, which at…
Refractory angina is a chronic disorder, which accompanies coronary artery disease. Patients experience lower physical activity and require support of…
Huntington’s disease is an obvious target for gene therapies. Caused by long repeats in a single specific gene and the…
A year ago, we covered preliminary results of ultra-personalized gene therapy for a rare form of Batten disease. Team behind the treatment…