Blind mice see again af­ter gene ther­apy

The Press and Journal (Inverness) - - NEWS -

blind­ness oc­cur due to the loss of mil­lions of light-sen­si­tive pho­tore­cep­tor cells that func­tion like cam­era sen­sors.

The re­search in­volved re­pro­gram­ming sur­viv­ing non-light sen­si­tive cells in the retina to con­vert them into new pho­tore­cep­tors.

A virus was used to carry the gene for the light-sen­si­tive pro­tein melanopsin into the cells’ DNA.

The tech­nique was tested on lab­o­ra­tory mice blinded by the de­gen­er­a­tive dis­ease re­tini­tis pig­men­tosa (RP), the most com­mon cause of vi­sion loss in young peo­ple.

Clear ev­i­dence of re­stored sight was seen 13 months af­ter the virus was in­jected into the an­i­mals’ eyes. Their pupil re­flexes showed they re­sponded to light, and they were able to per­form tasks re­quir­ing ba­sic im­age recog­ni­tion.

Lead author Dr Sa­man­tha de Silva, from the Nuffield Lab­o­ra­tory of Oph­thal­mol­ogy at Ox­ford Uni­ver­sity, said: “There are many blind pa­tients in our clin­ics and the abil­ity to give them some sight back with a rel­a­tively sim­ple ge­netic pro­ce­dure is very ex­cit­ing. Our next step will be to start a clin­i­cal trial to as­sess this in pa­tients.”

Her group is cur­rently run­ning two tri­als of other gene ther­a­pies for the in­her­ited blind­ness dis­or­ders choroi­deremia and Xlinked re­tini­tis pig­men­tosa.

Both of these took two to three years to com­plete the jour­ney from lab to clinic, she said.

Dr de Silva said: “We hope that a hu­man melanopsin gene ther­apy clin­i­cal trial would be achiev­able along the same timescale.”

The sci­en­tists added: “These re­sults sug­gest that this ap­proach may be clin­i­cally use­ful in vi­sion restora­tion in pa­tients with end-stage RP.”

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