Table 1.
Multi-year persistence of clotting factor expression following AAV gene therapy in large animals and in humans
IHC indicates immunohistochemistry; and FISH: fluorescence in situ hybridization.
* Adjunctive bortezomib ± dexamethasone administered at the time of AAV vector administration. Follow-up results updated via personal communication from Clinton Lothrop.
† Length of follow-up updated. Oral presentation by A.C. Nathwani at the American Society of Cell and Gene Therapy meeting in New Orleans, LA, May 2015.
