New NIH vi­ral vec­tor flips the script on sick­le cell dis­ease gene ther­a­py

Re­searchers at the NIH have rolled out a new ve­hi­cle for sick­le cell gene ther­a­py with high­er speeds and bet­ter horse­pow­er, po­ten­tial­ly al­low­ing vast­ly more ef­fi­cient gene trans­fer and a much larg­er car­ry­ing ca­pac­i­ty. The best part? Un­like cur­rent sick­le cell gene ther­a­py mod­els, the NIH one doesn’t have to dri­ve in re­verse.

In mice and mon­keys, the new ve­hi­cle was up to 10 times more ef­fi­cient and had a car­ry­ing ca­pac­i­ty – the amount of DNA it can haul – of up to 6 times that of the con­ven­tion­al vec­tors cur­rent­ly de­ployed in gene ther­a­py tri­als across the coun­try. Most no­tably, the new vec­tor can read the ther­a­peu­tic gene se­quence for­ward rather than read­ing them back­ward — a counter-in­tu­itive trick re­searchers had used to over­come long-run­ning bar­ri­ers to gene ther­a­py but which sac­ri­ficed ef­fi­cien­cy. The re­sults were pub­lished open ac­cess in Na­ture Com­mu­ni­ca­tions.

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