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APowerfulNewWaytoEditDNAMARCH3,2014
Matter:InShortLivedFish,SecretstoAgingFEB.27,2015
Ethicists,fordecades,havebeenconcernedaboutthedangersofalteringthehumangermlinemeaning
tomakechangestohumansperm,eggsorembryosthatwilllastthroughthelifeoftheindividualandbe
passedontofuturegenerations.Untilnow,theseworrieshavebeentheoretical.Butatechniqueinventedin
2012makesitpossibletoeditthegenomepreciselyandwithmuchgreaterease.Thetechniquehasalready
beenusedtoeditthegenomesofmice,ratsandmonkeys,andfewdoubtthatitwouldworkthesameway
inpeople.
Thetechniqueholdsthepowertorepairorenhanceanyhumangene.Itraisesthemostfundamentalof
issuesabouthowwearegoingtoviewourhumanityinthefutureandwhetherwearegoingtotakethe
dramaticstepofmodifyingourowngermlineandinasensetakecontrolofourgeneticdestiny,whichraises
enormousperilforhumanity,saidGeorgeQ.Daley,a
stemcell
expertatBostonChildrensHospitalanda
memberofthegroup.
ThebiologistswritinginSciencesupportcontinuinglaboratoryresearchwiththetechnique,andfewifany
scientistsbelieveitisreadyforclinicaluse.AnysuchuseistightlyregulatedintheUnitedStatesand
Europe.Americanscientists,forinstance,wouldhavetopresentaplantotreatgeneticdiseasesinthe
humangermlinetotheFoodandDrugAdministration.
Thepapersauthors,however,areconcernedaboutcountriesthathavelessregulationinscience.They
urgethatscientistsshouldavoidevenattempting,inlaxjurisdictions,germlinegenomemodificationfor
clinicalapplicationinhumansuntilthefullimplicationsarediscussedamongscientificandgovernmental
organizations.
Thoughsuchamoratoriumwouldnotbelegallyenforceableandmightseemunlikelytoexertglobal
influence,thereisaprecedent.In1975,scientistsworldwidewereaskedtorefrainfromusingamethodfor
manipulatinggenes,therecombinantDNAtechnique,untilruleshadbeenestablished.
Weaskedatthattimethatnobodydocertainexperiments,andinfactnobodydid,tomyknowledge,said
Dr.Baltimore,whowasamemberofthe1975group.Sothereisamoralauthorityyoucanassertfromthe
U.S.,andthatiswhatwehopetodo.
RecombinantDNAwasthefirstinaseriesofeverimprovingstepsformanipulatinggeneticmaterial.The
chiefproblemhasalwaysbeenoneofaccuracy,ofeditingtheDNAatpreciselytheintendedsite,sinceany
offtargetchangecouldbelethal.Tworecentmethods,knownaszincfingersandTALeffectors,cameclose
tothegoalofaccurategenomeediting,butbotharehardtouse.The
newgenomeeditingapproach
was
inventedbyJenniferA.DoudnaoftheUniversityofCalifornia,Berkeley,andEmmanuelleCharpentierof
UmeaUniversityinSweden.
Theirmethod,knownbytheacronymCrisprCas9,cooptsthenaturalimmunesystemwithwhichbacteria
remembertheDNAofthevirusesthatattackthemsotheyarereadythenexttimethosesameinvaders
appear.Researcherscansimplyprimethedefensesystemwithaguidesequenceoftheirchoiceanditwill
thendestroythematchingDNAsequenceinanygenomepresentedtoit.Dr.Doudnaistheleadauthorof
theSciencearticlecallingforcontrolofthetechniqueandorganizedthemeetingatwhichthestatementwas
developed.
Thoughhighlyefficient,thetechniqueoccasionallycutsthegenomeatunintendedsites.Theissueofhow
muchmistargetingcouldbetoleratedinaclinicalsettingisonethatDr.Doudnasgroupwantstosee
thoroughlyexploredbeforeanyhumangenomeisedited.Scientistsalsosaythatreplacingadefectivegene
withanormalonemayseementirelyharmlessbutperhapswouldnotbe.Weworryaboutpeoplemaking
changeswithouttheknowledgeofwhatthosechangesmeanintermsoftheoverallgenome,Dr.Baltimore
said.Ipersonallythinkwearejustnotsmartenoughandwontbeforaverylongtimetofeel
comfortableabouttheconsequencesofchangingheredity,eveninasingleindividual.