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16 February 2017

A new approach to haemophilia B treatment is showing promise in its animal testing phase, researchers have reported.

Researchers from the University of Pennsylvania School and the University of Florida in the USA looked for methods of preventing haemophilia B patients from developing antibodies against coagulation factor IX, usually needed to prevent uncontrolled bleeding.

Professor Henry Daniell and colleagues explained in the journal Molecular Therapy recently that using general immune suppression is problematic.

So, 'to address this urgent unmet medical need, we delivered antigen bioencapsulated in plant cells to haemophilia B dogs', they write.

Specifically, this involved genetically modified lettuce leaf cells that express elements of coagulation factor IX. When inserted into the DNA of the plant's chloroplasts, it allows bodily cells to tolerate rather than block coagulation factor IX.

'Successful scale up of production and development of a storable edible plant product allowed us to evaluate the approach in a large animal model of haemophilia of similar size as paediatric patients that are likely candidates for oral tolerance,' the team reports.

'Coagulation times were markedly shortened in orally tolerised treated dogs, in contrast to control dogs that formed high-titre antibodies,' they write, adding that no side effects were detected after giving the animals this treatment for more than 300 days.

Professor Daniell said: 'The results were quite dramatic. We corrected blood clotting time in each of the dogs and were able to suppress antibody formation as well. All signs point to this material being ready for the clinic.'

Source: Herzog, R. W. et al. Oral Tolerance Induction in Hemophilia B Dogs Fed with Transplastomic Lettuce. Molecular Therapy 22 December 2016; doi: 10.1016/j.ymthe.2016.11.009

Link: http://www.cell.com/molecular-therapy-family/molecular-therapy/fulltext/S1525-0016(16)45392-X


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