Gene & cell therapies
Gene therapy evidence: distinguish early response from durable benefit
A one-time administration still requires long-term evidence about effectiveness, late adverse events and who remains represented in follow-up.
Reader perspective: Researchers
Clinical research
Rare-disease trial endpoints: measure a change that matters
Small populations make endpoint selection more demanding, not less; connect measurement quality, natural history and patient relevance.
Genomic testing
Pseudogene interference: establish which DNA your assay measured
CYP21A2 illustrates how similar DNA sequences and complex gene structures challenge genetic analysis—and why research methods need locus-specific validation.