Science
Ge Zhang, Honglin Zheng, Qiang Li, Taiqi Zhao, Teng Li. Recording results from single-patient trials. Science. 2026;394(6819):38–39. https://doi.org/10.1126/science.aek9583
Recording results from single-patient trials
This Science Letter argues that public trial registries and preclinical publications should retain a current, linked account of directly related human protocols and major outcomes in individualized genetic therapies. It proposes reporting practices, rather than presenting a new clinical trial or establishing treatment causality.
Single-patient describes the research context discussed in the Letter. It is not an observed n=1 dataset produced by these authors; no sample total is assigned.
How should public records preserve major human outcomes when individualized genetic therapy research involves a single patient?
The Letter responds to previously reported individualized base-editing research and a public registry entry that, according to the Letter, remained out of date after the reported participant death. The authors argue that an individual outcome may constitute the available human evidence in a single-patient study, making a current public record important to families, clinicians, ethics committees and other researchers. They distinguish confidential or regulatory safety reporting from public trial registries and journal publications. Their proposals include disclosure of directly related human protocols in preclinical submissions, updating those disclosures before acceptance, recording treatment-emergent deaths and life-threatening events separately from causal assessment, linking preclinical papers with trial records, and adding dated notices when major human outcomes occur after publication. These are the Letter's reporting and governance arguments, not a newly conducted experiment, demonstrated clinical benefit or determination of responsibility.
The opening paragraph contrasts the previously reported human outcome with the registry and preclinical publication as described by the Letter. This is an attributed reporting-gap example, not an independently reconstructed clinical investigation.
Source locator: Print p. 38, right column, Letter paragraph 1
The authors explain why families, clinicians, ethics committees and other researchers may rely on a registry when considering continued human testing and emerging risks.
Source locator: Print p. 38, right column, paragraph 2, continuing at p. 39, left column
Safety reports, registries and journal articles serve different purposes and update on different schedules; a report reaching regulators or ethics committees does not by itself ensure that public records are current.
Source locator: Print p. 39, left column, Letter paragraph 3
The authors propose disclosure of directly related human protocols before preclinical paper acceptance, registry fields for treatment-emergent deaths and life-threatening events, separation of event occurrence from suspected or confirmed causality, cross-links between records, and dated notices after publication.
Source locator: Print p. 39, left column, Letter paragraph 4; reference 3 provides the cited registration-standard context
The concluding paragraph sets an explicit boundary: the reporting proposals connect claims of clinical promise to subsequent human experience, while leaving causal and responsibility determinations unresolved.
Source locator: Print p. 39, left column, Letter paragraph 5, immediately before the author line
Cite the Letter's reporting rationale and the consequences of limited human evidence, with the discussed case attributed to prior sources.
Cite the proposed disclosure, registry, linking and notice practices; implementation effectiveness was not measured.
Cite its explicit reporting boundary, without treating the Letter as a safety or responsibility adjudication.
Evidence: KF5
| Row ID | Component | Context | Finding | Evidence Level | Scope | Source Locator | Evidence refs |
|---|---|---|---|---|---|---|---|
| evidence-science-records | Current public records | Reported registry-publication gap, attributed to the Letter and its cited sources. | Keep directly related human protocols and major outcomes current in public registries. | Letter argument | Reporting rationale, not an independent clinical investigation. | Print p. 38, right column, Letter paragraph 1 | KF1 |
| evidence-science-visibility | Human evidence visibility | Readers may rely on the public entry when only an individual outcome is available. | Treat a current public record as consequential when an individual outcome may be the available human evidence. | Letter argument | Evidence visibility, not a new cohort or clinical sample. | Print p. 38, right column, paragraph 2, continuing at p. 39, left column | KF2 |
| evidence-science-channels | Reporting channels | Oversight reports and public records can update on different schedules. | Distinguish safety reporting to oversight bodies from the publicly accessible account of translation. | Letter argument | Reporting-channel distinction, not a quantified audit. | Print p. 39, left column, Letter paragraph 3 | KF3 |
| evidence-science-proposals | Coordinated updates | Proposals connect protocol disclosure, serious outcomes, registry links and later notices. | Coordinate protocol disclosure, serious-outcome reporting, cross-links and dated publication updates. | Letter proposal | Proposed practices, not tested effectiveness or treatment causality. | Print p. 39, left column, Letter paragraph 4; reference 3 provides the cited registration-standard context | KF4 |
| evidence-science-boundary | Causality boundary | The conclusion sets limits on what public notices establish. | Use public notices to preserve the human experience without assigning blame or settling causality. | Explicit Letter boundary | Documentation of experience, not adjudication. | Print p. 39, left column, Letter paragraph 5, immediately before the author line | KF5 |
The authors describe a gap between a previously reported human outcome and the registry or preclinical publication, and argue that directly related human protocols and major outcomes should remain visible in current public records. The case is contextual evidence from other sources, not a new study in this Letter.
Evidence: KF1
An individual outcome may be the available human evidence. The Letter argues that families, clinicians, ethics committees and other researchers can rely on the public entry when judging continued testing and emerging risks.
Evidence: KF2
The Letter distinguishes safety reports, trial registries and journal articles because their purposes and update schedules differ. Clinical information reaching an oversight body can still be absent from the public account of translation.
Evidence: KF3
They propose disclosure and updating of directly related human protocols before preclinical manuscript acceptance, reporting of treatment-emergent deaths and life-threatening events separately from causal assessment, links between preclinical papers and trial records, and dated notices for major later human outcomes.
Evidence: KF4
No. Its conclusion explicitly separates documenting subsequent human experience from assigning blame or settling causality. Its proposals concern reporting and governance, with no new clinical experiment or quantified treatment effect.
Evidence: KF5
Study-level author-maintained evidence summary. Not participant-level data and not a ready-to-pool meta-analysis dataset. For formal citation, use the original article DOI.
Download study-level evidence CSV (UTF-8)Formula safety: cells starting with =, +, -, @ (after leading whitespace), or tab/CR/LF are prefixed with an apostrophe. Treat all cells as text; remove only that safety prefix when recovering the original string.
These records identify the same original work; database inclusion is not evidence of scientific validity or clinical benefit.