Using CRISPR / Cas9, the Hamon Center team was able to correct the genetic defect in a mouse model of DMD and thus prevent the development of features of the disease, which in boys
causes progressive muscle weakness and degeneration, often along with breathing and heart complications.
The illness
causes progressive muscle weakness and brain damage.
Not exact matches
His parents confirmed that Charlie had died in a hospice yesterday after a long battle with an extremely rare genetic condition
causing progressive brain damage and
muscle weakness.
It is characterized by the wasting of skeletal
muscles and
caused by
progressive degeneration of nerve cells in the spinal cord; the disease leads to increasing muscular
weakness, atrophy and premature death due to respiratory problems.
Spinal muscular atrophy is a genetic disease that
causes the degeneration of spinal cord motor neurons and leads to
progressive muscle weakness, atrophy and inability to walk or sit, and breathing difficulties.
It
causes progressive weakness in
muscles of the face, arms, legs, and around the shoulders and chest.