How does gene replacement therapy work?How does gene replacement therapy work?

Gene replacement therapy is being studied as a treatment option for genetic diseases, and we want to help you understand

Bringing Hope to Children Through a Revolution in Gene TherapyBringing Hope to Children Through a Revolution in Gene Therapy

In pediatric neurology, many diseases are caused by absent or mutated genes. The genomic revolution has allowed scientists to sequence

Clinical, biochemical, and genetic features of four patients with ECHS1Clinical, biochemical, and genetic features of four patients with ECHS1

Short‐chain enoyl‐CoA hydratase (SCEH or ECHS1) deficiency is a rare inborn error of metabolism caused by biallelic mutations in the