The Critical Need to Consolidate All Gene Therapy Data in Haemophilia
Bibliographic record
Abstract
To the Editor: Gene therapy for haemophilia has been in development for several decades, with the first clinical attempts initiated in the 1990s. These early studies, including six initial trials, laid the foundation for subsequent systemic liver-directed adeno-associated virus (AAV) trials that ultimately led to regulatory approvals. A pivotal milestone came in 2011 with the publication by Nathwani et al., which demonstrated the first successful systemic AAV8-FIX gene therapy for haemophilia B, achieving sustained therapeutic factor IX levels with immune modulation using prednisolone. [...]
Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.
How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.046 | 0.097 |
| Meta-epidemiology (narrow) | 0.001 | 0.001 |
| Meta-epidemiology (broad) | 0.005 | 0.002 |
| Bibliometrics | 0.005 | 0.004 |
| Science and technology studies | 0.002 | 0.006 |
| Scholarly communication | 0.014 | 0.025 |
| Open science | 0.004 | 0.007 |
| Research integrity | 0.012 | 0.024 |
| Insufficient payload (model declined to judge) | 0.023 | 0.010 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".