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Gene Therapy & CRISPR Cas 9 [PPMD's 2016 Connect Conference]

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Gene Therapy isn't new in the world of Duchenne. In fact, we have had gene therapy presentations at the Connect Conference the past 20 years. However, we are now at a point where gene therapy is closer than ever before and a potential treatment opportunity we see emerging quickly. But what is gene therapy? What is CRISPR Cas 9? How are they similar? Different? What questions do we need think about, to ask, when considering these opportunities? This panel discussion at PPMD's 2016 Connect Conference brought together many of the researchers, industry partners, and others working in this exciting area.

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duchenne dmd duchenne muscular dystrophy gene therapy dystrophin crispr cas9 RNA chromosome gene genetic code heredity nucleic acid DNA Gene medicine remedy cure healing analysis harm hurt attitude behaviorism mental make-up mental processes personality study psych science of the mind therapy way of thinking where head is at antidote assistance biologic corrective counteractant counteraction counteractive counteragent countermeasure counterstep cure-all drug elixir fix improvement medicament medicant panacea pharmaceutical pharmacon physic pill quick fix redress relief restorative support treatment diet doctoring hospitalization medication operation prescription regimen surgery therapeutics aversion therapy behavioral therapy conditioning counseling reinforcement retraining reward system 12-step group Alcoholics Anonymous Weight Watchers encounter group family friends morale boosters safety net self-help group support system psychology behavior modification support group psychoanalysis Therapy crispr C/A central air central air conditioning climate control climatization air conditioning air cooling ppmd 2016 attach relate join bridge affix span wed couple unite tag yoke equate fasten cohere marry ally tag on tie in detach loosen sever divide unlink abut approximate be adjacent to border butt communicate connect lie lie beside link neighbor touch verge add annex append combine interconnect align balance bring into line calibrate correct fine-tune fit fix focus grind improve improve accommodate mend overhaul polish put in working order readjust rectify regulate renovate repair service set sharpen square tighten troubleshoot tune up amalgamate associate band together come aboard confederate form connection go partners hook up incorporate line up plug into team up throw in with tie up adjoin appropriate hitch on hitch up hook on slap on subjoin tack on take on take over affect allude appertain be applicable be pertinent bear upon concern involve pertain refer regard suit adjust affiliate apply connects CONNECT Connects conference Conference Duchenne DMD dmd sinewy burly sturdy brawny stout robust wiry hefty husky lusty mighty strong tiger tough hulky ripped ropy small thin weak beastly bestial bodily brute brutish corporeal earthly earthy feral fleshy mammalian muscular natural physical sensual untamed wild zoological able-bodied active energetic fit powerful strapping vigorous athletic beefcake beefy big bruising bulky gorillalike hulking hunk portly stocky thickset well-built coarse fibroid hairy pulpy stalky threadlike tissued veined woody able acclimatized capable enduring firm fresh hale hardened healthy hearty in fine fettle in good condition in good shape indefatigable inured physically fit resistant rugged seasoned solid sound stalwart staunch substantial tenacious unflagging well all right blooming bright-eyed bushy-tailed chipper flourishing full of life hardy healthful in fine feather in the pink lively normal potent restored rosy-cheeked safe and sound trim unimpaired virile whole animal fibrous gene therapy RNA chromosome gene genetic code heredity nucleic acid DNA Gene medicine remedy cure healing analysis harm hurt attitude behaviorism mental make-up mental processes personality study psych science of the mind therapy way of thinking where head is at antidote assistance biologic corrective counteractant counteraction counteractive counteragent countermeasure counterstep cure-all drug elixir fix improvement medicament medicant panacea pharmaceutical pharmacon physic pill quick fix redress relief restorative support treatment diet doctoring hospitalization medication operation prescription regimen surgery therapeutics aversion therapy behavioral therapy conditioning counseling reinforcement retraining reward system 12-step group Alcoholics Anonymous Weight Watchers encounter group family friends morale boosters safety net self-help group support system psychology behavior modification support group psychoanalysis Therapy atl help heals amazing trannie male twat tosser cross-dresser loser middle aged whiskers nose touch cartilage process and push. psychotherapy talking fo sho abandonment pain crispr
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