The hemophilias--from royal genes to gene therapy.

By P. Mannucci, E. Tuddenham. New England Journal of Medicine, 2001

1,092 citations recorded by the source corpus.

Subfields: Hematology, Molecular Biology

View Paper: https://www.semanticscholar.org/paper/a0b617587e7110edcf2fdcbc7a5a7afae30adcf5

How researchers see this paper

The researchers whose trusted networks rank it highest, strongest advocate first. Every researcher gets a different order.

1. S. Al-Zaidy
2. J. Mendell
3. L. Rodino-Klapac
4. Kimberly Goodspeed
5. S. Gray
6. Stephanie M Salabarria
7. B. Byrne
8. J. Tremblay

Citations in the corpus

• Current Clinical Applications of In Vivo Gene Therapy with AAVs (2020, background): “Bleeding sequelae correlate with plasma FVIII/FIX activity (FVIII:C/FIX:C), such that spontaneous hemorrhage occurs in severe HA/HB (FVIII/FIX:C <1% of normal), less commonly in moderate HA/HB (FVIII/FIX:C 1%–5% of normal), and typically only following trauma in mild HA/HB (FVIII/FIX:C >5%–40% of no”