Frommiraclecurestosuperbabies:thefacebehindthegenomeeditingmania

By:AlexiyNikiforov&MichelleValkanas

http://news.berkeley.edu/2015/11/12/crispr-cas9-gene-editing-check-three-times-cut-once/

Regardlessofyourscientificbackground,noonecandenytheimpactCRISP-Cas9willhaveonhumanity’sfuture.CRISPR-Cas9,originallydiscoveredinbacteriaasadefensemechanismagainstviruses,isnowbeingconsideredasasystemforgenomeediting.OntheforefrontofthistechnologyisJenniferDoudnaaProfessorofMolecularandCellBiologyandofChemistryfromtheUniversityofCalifornia,BerkeleywhowasoneoftheoriginalauthorsofthepaperfirstdescribingCas9.Sinceherdiscoverywasfirstpublishedin2012,ithasabeenwhirlwindofsuccessandhonors,includingbeingnamedinTIMEMagazine’s100MostInfluentialPeopleof2015.TheUniversityofPittsburghalsopresentedherwiththe2016DicksonPrizeinMedicinethisyearattheirconferenceentitledScience2016:GameChangers.Withthisnewlyfoundsuccessthemorallineappearstobebecomingblurred.Frompatentwarstosuperbabiesandmagicalmushrooms,thedebateoverthistechnologyhasspreadworldwide.

http://rna.berkeley.edu/crispr.htmlCRISPRarethehallmarksofacquiredimmunityinbacteria.TheCRISPRloci,consistingofCasgenesand“spacers”,areabletoadapttoforeignDNAinjectedintothecellandstoreasadefensemechanismforfutureattacks.ItwasthendiscoveredthatCas9wasabletobeprogrammedtocutspecifictargetedDNA.ThereareavarietyofoptionsusingCas9editing.Itisextremelyaccurateinrecognizingnucleicacidbasepairsallowingittofunctionasaguidedsystem.ItalsoallowsforprogrammableDNAmanipulationinbothplantsandanimals.

http://www.nature.com/nature/journal/v495/n7439/full/495050a.html

Thoughthisdiscoverycanleadtoanarrayofusefultechniques,itsprominentfunctionisintherapeuticapplications.Withthatcomeschallenges,includingaproperdeliverysystemforeachspecificlocationinthebody,controllingthepathwaysofDNArepairthatisrespondingtothecleavingofthesite,andthenumerousethicalconcernsinvolved.ThroughthediscoveryofCas9thepossibilitiesareendless,butJenniferDoudnadidnotseemtowanttalkaboutanyofthem.Thoughherhandmayhavebeentiedalittlebit,shecouldhaveatleastpresentedtheissuesathand.Insteadwegotashortblipaboutamagicalmushroomthatdoesnotturnbrownandapictureofababy.

http://cdn.static-economist.com/sites/default/files/cf_images/images-magazine/2015/08/22/LD/20150822_LDP001_facebook.jpgWhydidn’tshegetonstageandboastaboutthepossiblecureofSickleCellAnemia,isitbecauseshewouldthenhavetodiscusstheelephantintheroom?DoudnaherselffearsthefutureasshesharedwithNatureinaninterviewlastyear,“[I]ponderedhowIwouldfeelifthenextreportertocontactmewantedtoknowaboutgenome-editingworkinvolvinghumanembryos.‘Howlongwillitbebeforesomeonetriesthisinhumans?’Iwonderedaloudtomyhusbandoverbreakfastthenextday.”Shegoesontosay,“thesediscussionshavepushedmefaroutsidemyscientificcomfortzone”.JenniferDoudnahasstumbledontosomethingfarbiggerthenherself,somethingthatcouldchangethecourseofhumanevolutionforever!Nooneknowswhatthefutureholds,maybeonedaywewillbepickingourchildrenoutofacatalog.