{"schemaVersion":"maha-epistemic/1.0","evidencePolicyVersion":"mps/0.1","recordId":"urn:maha:record:ex-vivo-genome-editing-workflow","canonicalPath":"/knowledge/synthetic-biology/methods/ex-vivo-genome-editing-workflow","contentHash":"sha256:835c473a58c8114c35a25c88d3e618e1f2954e0555c8c29bd0d89db232111877","generatedAt":"2026-08-30T16:02:46.045Z","publicationDecision":{"recordId":"urn:maha:record:ex-vivo-genome-editing-workflow","publicEligible":true,"evaluatedAgainst":"maha-epistemic/1.0","reasons":[]},"claims":[{"id":"urn:maha:claim:ex-vivo-genome-editing-workflow","scope":"The constructs, biological systems, protocols, assays, datasets, and comparisons reported in CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia.","boundary":"The workflow’s risks and outcomes are indication-, cell-product-, conditioning-, manufacturing-, participant-, and follow-up-specific.","claimKind":"empirical-claim","sourceIds":["source-frangoul-crispr-2021"],"statement":"The cited report describes collection, ex-vivo CRISPR-Cas9 editing, conditioning, transplantation, and early follow-up for two participants.","replication":{"asOfDate":"2026-08-24","assessment":"This candidate records one bounded source package. Independent replications and contradictory results must be compiled separately before evidence maturity is upgraded.","independentReplicationCount":null},"uncertainty":{"kind":"qualitative","statement":"There is no universal effect estimate for this method; numerical results remain attached to the source experiment, biological system, assay, and analysis choices."},"evidenceMaturity":"single-study"}],"sources":[{"id":"source-frangoul-crispr-2021","url":"https://www.nejm.org/doi/full/10.1056/NEJMoa2031054","title":"CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia","rights":{"note":"Maha paraphrases the source-level result and links to the version of record; no article passage is reproduced.","basis":"citation-with-paraphrase","quotationUsed":false},"authors":["Haydar Frangoul","David Altshuler","M. Domenica Cappellini","Yi-Shan Chen","et al."],"boundary":"Two early cases do not establish population-level safety, durability, comparative effectiveness, or general readiness of genome editing.","publisher":"The New England Journal of Medicine","establishes":"The report describes ex-vivo CRISPR-Cas9 editing, conditioning, transplantation, and early clinical outcomes for two participants under a specific investigational protocol.","identifiers":[{"value":"10.1056/NEJMoa2031054","scheme":"doi"}],"publishedAt":"2020-12-05","exactLocator":"Abstract; Methods; Results; figures and tables for the two reported participants; supplementary protocol.","conflictsOfInterest":"The study was sponsored by CRISPR Therapeutics and Vertex Pharmaceuticals; author relationships are disclosed in the article."}],"reviewEvents":[{"scope":"source-fidelity","verdict":"approve","reviewId":"epireview_6aa0615fc7604a609635eda707393ac9","rationale":"Ex-vivo genome-editing workflow binds claim urn:maha:claim:ex-vivo-genome-editing-workflow only to CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia at NEJM DOI 10.1056/NEJMoa2031054, Summary; Patient Demographics and Outcomes; Discussion. The audit inspected version-of-record at declared inspected location depth and recorded subject and claim support; the claim remains limited to “The constructs, biological systems, protocols, assays, datasets, and comparisons reported in CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia.”. This decision applies only to record urn:maha:record:ex-vivo-genome-editing-workflow at sha256:3e76f32cc4d0ff6e901f3aeecd576f89bfa43edf09be22e618256a3a0fcc9120 and does not certify truth, external endorsement, independent reproduction, or fitness for use.","reviewedAt":"2026-08-30T16:02:01.089Z","reviewerId":"expert_maha-internal-editorial-scale-v1","reviewMethod":"Each criterion is recomputed from the exact record, its inspected alignment audit, source identity, exact locator, rights basis, claim scope, boundary, uncertainty, replication status, prohibited inferences, and revision digest.","reviewerKind":"internal-editorial","reviewerRole":"AI-assisted record-specific review of inspected source identity, exact locator, bounded claim, uncertainty, non-claims, rights basis, and exact revision. This is not an external subject-matter credential.","targetSha256":"sha256:3e76f32cc4d0ff6e901f3aeecd576f89bfa43edf09be22e618256a3a0fcc9120","supersedesReviewId":null,"reviewerProfileVersion":1},{"scope":"domain-fidelity","verdict":"approve","reviewId":"epireview_6707ebbe804a4c9da88ac3561c66bba1","rationale":"Ex-vivo genome-editing workflow remains within domain synthetic-biology. Its mechanism or method is the bounded proposition “The cited report describes collection, ex-vivo CRISPR-Cas9 editing, conditioning, transplantation, and early follow-up for two participants.”; the record does not transfer that proposition beyond The workflow’s risks and outcomes are indication-, cell-product-, conditioning-, manufacturing-, participant-, and follow-up-specific. This decision applies only to record urn:maha:record:ex-vivo-genome-editing-workflow at sha256:3e76f32cc4d0ff6e901f3aeecd576f89bfa43edf09be22e618256a3a0fcc9120 and does not certify truth, external endorsement, independent reproduction, or fitness for use.","reviewedAt":"2026-08-30T16:02:01.157Z","reviewerId":"expert_maha-internal-editorial-scale-v1","reviewMethod":"Each criterion is recomputed from the exact record, its inspected alignment audit, source identity, exact locator, rights basis, claim scope, boundary, uncertainty, replication status, prohibited inferences, and revision digest.","reviewerKind":"internal-editorial","reviewerRole":"AI-assisted record-specific review of inspected source identity, exact locator, bounded claim, uncertainty, non-claims, rights basis, and exact revision. This is not an external subject-matter credential.","targetSha256":"sha256:3e76f32cc4d0ff6e901f3aeecd576f89bfa43edf09be22e618256a3a0fcc9120","supersedesReviewId":null,"reviewerProfileVersion":1},{"scope":"boundary-adequacy","verdict":"approve","reviewId":"epireview_d9d3eb8c4ad24097a686ddc6c705944c","rationale":"Ex-vivo genome-editing workflow retains uncertainty “There is no universal effect estimate for this method; numerical results remain attached to the source experiment, biological system, assay, and analysis choices.” and replication assessment “This candidate records one bounded source package. Independent replications and contradictory results must be compiled separately before evidence maturity is upgraded.”. Its non-claims and 2 prohibited inference(s) remain attached to every reuse. This decision applies only to record urn:maha:record:ex-vivo-genome-editing-workflow at sha256:3e76f32cc4d0ff6e901f3aeecd576f89bfa43edf09be22e618256a3a0fcc9120 and does not certify truth, external endorsement, independent reproduction, or fitness for use.","reviewedAt":"2026-08-30T16:02:01.225Z","reviewerId":"expert_maha-internal-editorial-scale-v1","reviewMethod":"Each criterion is recomputed from the exact record, its inspected alignment audit, source identity, exact locator, rights basis, claim scope, boundary, uncertainty, replication status, prohibited inferences, and revision digest.","reviewerKind":"internal-editorial","reviewerRole":"AI-assisted record-specific review of inspected source identity, exact locator, bounded claim, uncertainty, non-claims, rights basis, and exact revision. This is not an external subject-matter credential.","targetSha256":"sha256:3e76f32cc4d0ff6e901f3aeecd576f89bfa43edf09be22e618256a3a0fcc9120","supersedesReviewId":null,"reviewerProfileVersion":1},{"scope":"rights-and-locator","verdict":"approve","reviewId":"epireview_f6dde2cf887c49ff9075f97a6d5a08d9","rationale":"CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia is identified by doi:10.1056/NEJMoa2031054, inspected at Abstract; Methods; Results; figures and tables for the two reported participants; supplementary protocol., and retained under citation-with-paraphrase. This approval binds only exact revision sha256:3e76f32cc4d0ff6e901f3aeecd576f89bfa43edf09be22e618256a3a0fcc9120. This decision applies only to record urn:maha:record:ex-vivo-genome-editing-workflow at sha256:3e76f32cc4d0ff6e901f3aeecd576f89bfa43edf09be22e618256a3a0fcc9120 and does not certify truth, external endorsement, independent reproduction, or fitness for use.","reviewedAt":"2026-08-30T16:02:01.286Z","reviewerId":"expert_maha-internal-editorial-scale-v1","reviewMethod":"Each criterion is recomputed from the exact record, its inspected alignment audit, source identity, exact locator, rights basis, claim scope, boundary, uncertainty, replication status, prohibited inferences, and revision digest.","reviewerKind":"internal-editorial","reviewerRole":"AI-assisted record-specific review of inspected source identity, exact locator, bounded claim, uncertainty, non-claims, rights basis, and exact revision. This is not an external subject-matter credential.","targetSha256":"sha256:3e76f32cc4d0ff6e901f3aeecd576f89bfa43edf09be22e618256a3a0fcc9120","supersedesReviewId":null,"reviewerProfileVersion":1}]}