By inserting a complete gene, the technique provides a one-step solution that overcomes the limitations of CRISPR gene editing technology.
Gene Therapy
The protein dystrophin, absent in people with muscular dystrophy, will subsequently begin to be produced by the additional genetic code.
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.
