A disease that children suffer from.
This news can change lives, a new gene therapy for muscular dystrophy is bringing incredible hope, not only does it promise to slow the progression of this devastating disease, but it also has the potential to reverse muscle damage already caused and the best tests in humans They are scheduled to begin within 2 years.
For those who don't know, DMD is a genetic disease that prevents the production of Dystrophin, a protein essential for maintaining muscle strength and integrity. Children with DMD usually begin to have mobility problems at 3 or 4 years of age. , the disease progresses rapidly leading to loss of the ability to walk in adolescence and unfortunately patients rarely survive beyond the age of 20.
As DMD is a genetic disease, it is an ideal target for gene therapy; however, the gene that encodes dystrophin is one of the most unknown, which makes it difficult to impact it in the viral vectors used to treat it.
insert healthy genes into cells, but now scientists at the University of Washington have developed an ingenious solution, they fragmented the dystrophin gene, loaded these fragments and a series of viral vectors and added instructions for muscle cells to reassemble the protein.
The results are very promising.
The results of the tests with mice were spectacular, they observed the production of large amounts of dystrophin again and noted significant physiological corrections in the animals, the progression of the disease was stopped and in some cases the muscle loss was reversed, other previous studies attempted to create more of dystrophin with some success, especially in dog trials, however, the new technique from the University of Washington showed even more promising results and as a bonus requires a lower dose of therapy, which means fewer side effects.
Researchers are optimistic that this revolutionary technique can be applied to other genetic diseases caused by mutations in large genes, promising to transform treatments and give new hope to thousands of families around the world.
