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