Genetic medicine can leave people with rare mutations behind. But there's new hope

A New Approach to Genetic Medicine Could Help More Patients

Genetic medicine can leave people with rare mutations behind. But there’s new hope.

Weighing cure for sick kids against troubling ethical questions — Harvard Gazette

Photos by Niles Singer/Harvard Staff Photographer Health Weighing cure for sick kids against troubling ethical questions Science Center talk outlines potential and risks of gene editing Clea Simon Harvard Correspondent April 28, 2025 4 min read If our differences are part of what make us human, do we have the right — or the responsibility — to change them? That question was at the crux of “…

Genetic medicine may not help people with rare mutations. But there's new hope

Patients with exceedingly rare mutations have fewer options and poorer prospects than those with more typical forms of these diseases.

Patients with rare mutations look to experimental gene therapies

Patients with mutations have less options than those with more typical forms of diseases, and many are pinning hopes on experimental gene therapies.

Genetic medicine can leave people with rare mutations behind. But there's new hope

People with diseases caused by rare mutations have fewer options and poorer prospects than other patients despite rapid recent advancements in the growing field of genetic science.