Gene therapy for repairing neurons
After gene therapy, the paralyzed children partially recovered their motor functions
The joint work of researchers led by Paul Wuh-Liang Hwu from the National Taiwan University Hospital and Barry Byrne from the University of Florida College of Medicine was published in Science Translational Medicine (Wuh-Liang Hwu et al., Gene Therapy for Aromatic l-Amino Acid Decarboxylase Deficiency).
The first phase of clinical trials involved four patients aged four to six years with a rare hereditary disease – decarboxylase deficiency of aromatic L-amino acids. In children suffering from this disease, the brain does not produce neurotransmitters dopamine and serotonin. Patients remain practically paralyzed and die in early childhood.
During treatment, doctors injected a virus with a working copy of the aromatic L-amino acid decarboxylase gene into the area of the patients' brain where dopamine and serotonin should be produced. For 16 months after treatment, one of the patients could stand on his own, other patients could sit without assistance from six months to 15 months. All children had the opportunity to move their heads, they began to gain weight, the level of dopamine and serotonin increased.
The parents of these children also noted that the children began to sleep better, their emotional stability and eye movement coordination improved.
"The participants in this study have the most severe of the known hereditary diseases associated with impaired motor ability. Only gene therapy could help them. Our research opens the door to the possibility of early treatment of neurological diseases with the help of gene therapy. We hope that the results of our work will be important for the treatment of Parkinson's disease and other common diseases," Byrne said in an interview with The Scientist (Gene Therapy for Brain Disease).
Now scientists plan to cure twice as many patients: four from Taiwan and four from America.
Portal "Eternal youth" http://vechnayamolodost.ru21.05.2012