Gensko zdravljenje, ki bi lahko spremenilo zdravljenje Huntingtonove bolezni

V svetu biotehnologije se vsake toliko časa pojavi trenutek, ki lahko na novo napiše pravila panoge. Večina poskusov preboja ostane na pol poti - pomanjkanje podatkov, zapleti pri preskušanju, nevzdržni razvojni modeli. Toda tokrat so razmere drugačne. Majhnemu nizozemskemu podjetju je uspelo nekaj, kar ni uspelo nobenemu drugemu razvijalcu: njegova eksperimentalna genska terapija je pokazala trajen biološki učinek na Huntingtonovo bolezen, smrtno nevrodegenerativno bolezen, za katero še ni odobrenega zdravljenja.

Z znanstvenega vidika gre za preboj, s tržnega pa za eksplozijo optimizma. Delnice podjetja so v enem samem dnevu (po objavi uspeha) poskočile za skoraj 300 %, saj so vlagatelji začeli ceniti ne le potencialno vrednost same terapije, temveč tudi dejstvo, da gensko zdravljenje možganskih bolezni ni več teoretičen koncept. Biotehnologija, ki je dolga leta trpela zaradi skepse in izgub, je nenadoma postala epicenter novega vala kapitala. Rezultat je izjemen: delnica se je od…

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