Duo solves mystery regarding FVIII

So Turner set about running controls to rule out mistakes. Were the antibodies interfering with one another? Was there any contamination? Was the microscope working correctly? Could the reading be a false-positive, an inadvertent result of another reaction she hadn’t expected?

“No. 1, I had to convince myself,” she said. “I am always skeptical if something is too easy. I have reviewed many, many papers, and I can always find what people do wrong.”

Once she had convinced herself, she and Dr Moake had to convince the paper’s referees. As she’d expected, they were skeptical. She said the bulk of the work over the past year involved doing a number of controls to remove any doubts about the findings.

In the end, the research confirmed that FVIII was made in both HUVECs and GMVECs. Moreover, Turner found that FVIII, like VWF, is both stored and secreted from WPBs.

Dr Moake said the discovery has clear implications for any future treatments that aim to repair genetic defects in patients with hemophilia A.

“Now that we recognize that FVIII is normally synthesized in endothelial cells and stored in Weibel-Palade bodies,” Dr Moake said, “those become the precise, most effective physiological targets for gene delivery.”