Researchers from San Raffaele Telethon Institute for Gene Therapy in Italy has uncovered a potential approach to improve the delivery of hemophilia gene therapy via the deception of the immune system.

The Challenge

Presently, a number of late-stage gene therapies for hemophilia use adeno-associated virus (AAV) as a vector to delivery a gene that expresses a functional coagulation factor.  Humans have already developed natural immunity against AAV, rendering the therapy ineffectiv

The Italian researchers were able to integrate the CD47, a protein, into the surface of lentiviral vectors (LVs) so they could avoid discovery and termination by the immune system. The researchers found that in tests with primates, the LVs transferred therapeutic genes to liver cells—the key hemophilia B human target—without causing toxicity.  The findings were reported in the journal Science Translational Medicine.

Lead Research/Investigator

Alessio Cantor, San Raffaele Telethon Institute for Gene Therapy

Source: STM Sciencemag

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