To help children with rare diseases, we must solve drug delivery

TL;DR - This Nature commentary argues that nanoparticle drug-delivery technologies could help make gene therapies viable for children with rare diseases and few treatment options.

• Effective delivery remains a central obstacle for pediatric gene therapy.
• Nanoparticles offer a potential mechanism for transporting therapies to target tissues.
• The provided excerpt does not specify particular nanoparticle designs, clinical results, or demonstrated efficacy.