(Picture credit: JIMMY WELCH)
In order to learn about a new drug therapy for treating amyotrophic lateral sclerosis (ALS), researchers in Spain have successfully produced a mouse embryo, through gene editing. It is believed to be the first such research on human stem cells.
The technique, which involves inserting a genetic construct directly into a cell with the help of two types of cells, was carried out at University of Barcelona’s Institute of Biochemistry and Molecular Biology (IBM/Initiative CIBM/Universitat Clube Prensa y de La Rioja).
A patient in Spain underwent a procedure to get the technique in May this year. Her doctor at University of Barcelona, Dr. Josep Martínez-Ribeiro, implanted the chimeric stem cells into the left and right lateral ventricle of her thigh. The patient has been diagnosed with spinal muscular atrophy, a condition in which movement of the leg are severely restricted because of an abnormal degeneration of muscle fibers.
After four or five days, the patients started to walk normally around their home. “They’re no longer tired, they run a little bit,” said Martínez-Ribeiro, whose patient is still undergoing treatment in Barcelona. He is confident that this kind of gene-editing will be useful for the treatment of other severe disorders.
The technique involves a combination of gene therapy and cell transplantation techniques, that are known to be safe and effective. Martínez-Ribeiro believes that this procedure, which he had never attempted before, is important because it will help researchers to improve the knowledge of cells using DNA, which was first used by the Greeks.
Gene therapy is a type of genetic manipulation that can turn cells into other types. Cells can be edited to change their genetic properties.
“The technique is based on using genes of one kind of cell and modifying them with the help of the cells of another kind,” explained Martínez-Ribeiro. He adds that it’s not easy because cells have to carry different genes. “This is not an uncommon problem,” he added.
The new treatment also requires the use of two types of cells to repair gene defect. Both cells are used to construct the new gene construct. Only the donor cells carry the defective gene.
“It’s the first time in a long time that a technique has been able to make an embryonic