The protein dystrophin, absent in people with muscular dystrophy, will subsequently begin to be produced by the additional genetic code.
Gene Therapy
Delivering a healthy copy of a gene to a person whose gene(s) are malfunctioning is known as gene therapy.
Researchers at Umeå University demonstrate that a novel gene treatment has significantly slowed down the disease development in a patient with an extremely severe type of ALS illness.
Of the six treated youngsters, five showed improvement in their hearing.
