Gene replacement therapy helps children overcome deadly illnesses

Replacing a defective gene with a healthy gene allows several children with rare hereditary diseases to have a typical life experience, such as going to school or living with their family. ..

To Alice Klein

Children with some rare genetic conditions, who once died in just a few years, can now have the typical expected lifespan with gene replacement therapy.

“We are treating children with fatal illnesses. It’s great.” Donald corn At the University of California, Los Angeles.

Korn and his colleagues are trying gene therapy (replacing the disease-causing version of the gene with a functioning version) for leukocyte adhesion deficiency type 1 (LAD-1).

This rare situation happens …