Hemophilia Gene Therapy: Caught Between a Cure and an Immune Response
Bibliographic record
Abstract
Gene therapy for the X-linked bleeding disorder hemophilia B'factor IX (FIX) deficiency'by means of in vivo gene transfer with adeno-associated viral (AAV) vectors has been in clinical trials for the past 16 years, cycling between partial successes in the clinic and further development in the laboratory. At the recent World Congress of the International Society on Thrombosis and Haemostasis (Toronto, Canada, June 2015), Paul Monahan and colleagues presented data from a patient who has achieved FIX levels of 20–25% that have been sustained for more than 6 months since undergoing hepatic gene transfer with an AAV8 vector (administered via peripheral vein).
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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 | 0.004 | 0.003 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.000 |
| Bibliometrics | 0.001 | 0.000 |
| Science and technology studies | 0.001 | 0.003 |
| Scholarly communication | 0.003 | 0.005 |
| Open science | 0.001 | 0.002 |
| Research integrity | 0.003 | 0.007 |
| Insufficient payload (model declined to judge) | 0.005 | 0.002 |
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".