Regulators, mount up.
basic_science · Level V
Where this comes from
- Record sourced from PubMed, PMID 39361768.
- Also identified by DOI 10.1126/science.ads5951.
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Abstract
Cryptic exons enable delivery of therapies only to sick neurons in a motor neuron disease.
Medical subject headings
- Exons
- Genetic Therapy
- Motor Neuron Disease
- Precision Medicine