Team Seek and Destry 3 Ideas Presentatin March 14, 2012 Marianne Lintz David McClelland Gabi Garcia Jenn Thrntn Hannah Kemptn Mentrs: Nahum and Lauren
Diabetes http://www.healthaidindia.cm/diabetes-care-in-india/mnitring-bld-sugar-level.html
Diabetes Imprtance: Type 1 Diabetes: beta-cells killed by wn immune system Lss f insulin prductin is devastating Lifelng, uncmfrtable treatment Disruptin f many rgan system functins due t uncntrlled bld sugar levels
Diabetes Idea: Intrduce rganisms in pancreas that target antigens released frm beta-cells by: Eating up antigens Using antibdies that mre cmpetitively bind t the antigens
Diabetes Impact: Dramatically reduce symptms Slw the nset f Diabetes Optimistically, eliminate the disease itself
Diabetes Cmpetitin: Traditinal symptm treatment Bld glucse level mnitring Insulin injectin (i.e. pump) Type 1 Diabetes has been cured in mice Gene therapy injectins (liver stem cells) Replaces, nt rescues, insulin-making cells Unclear whether permanent in humans
Diabetes Knwns: Mechanisms f Type 1 Diabetes Hw ne acquires Type 1 Diabetes Unknwns: Uncertainty f aut-immune respnse Pssibility f mutatins f induced rganism Hw t maintain ur synthetic system
Cystic Fibrsis http://www.uvm.edu/~animrsch/?page=milestne-cysticfibrsis.php
Cystic Fibrsis Imprtance: Nnfunctinal membrane in channel Fatal. Shrt life span (usually 20s) Many rgan systems are incredibly weak and dysfunctinal (i.e. respiratry, digestive) Often leads t ther cmplicatins, including high risk f infectin
Cystic Fibrsis Idea: Target Pseudmnas aeruginsa (PA) Causes lung infectin Mst cmmn killer f CF patients Induce apptsis f PA cells in lungs Intrduce sme rganism that can bind t the bacteria, fr example, and break dwn the cell wall
Cystic Fibrsis Impact: Over 50% CF patients have PA infectin Lengthen lifespan f patients Ease lifestyle Reduce the risk f infectin and early death
Cystic Fibrsis Cmpetitin: Currently n cure Antibitics fr infectins TOBI, Azithrmycin, Caystn Already n market fr fighting infectin-causing bacteria clnizing airways
Cystic Fibrsis Knwns: Cystic fibrsis is a painful and debilitating disease Unknwns: Frequency f injectins t destry PA cells Mnitring rganism (i.e. replicatin, mutatin, etc.)
Muscular Dystrphy http://www.faqs.rg/health/bdy-by-design-v1/the- Muscular-System-Design-parts-f-the-muscularsystem.html http://static.disabm.cm/cntent/images/articles/ cntent/22707.jpg
Muscular Dystrphy Imprtance: Debilitating illness that strikes dwn the muscular system Almst exclusively targets males Causes muscular system t weaken ver time and makes mvement difficult Usually require a wheelchair by early adlescence. Currently n cure
Muscular Dystrphy Fcus: Duchenne and Becker's DMD gene mutatin Similar prblem dealing with inadequate dystrphin Duchenne- n dystrphin is prduced Becker's- shrtened dystrphin is prduced Dystrphin is a prtein that acts as a spring t help prevent wearing away f muscles
Muscular Dystrphy Idea: Target dystrphin dysfunctinal prtein in tw majr types f MD Fllw up after detectin f inadequate dystrphin levels in skeletal muscles at infancy Untreated MD: Gene --> mrna --> prtein Treated MD: Gene --> mrna --> prtein Synthetic System --> mrna --^
Muscular Dystrphy Impact: Create enugh dystrphin in bdy at early age t hpefully prevent the disease's crippling manifestatin Stp lasting muscular damage Lengthen lifespan Thse with Duchenne MD usually live int their twenties, thse with Becker int their frties r abve.
Muscular Dystrphy Cmpetitin: Significant research and develpment is being made with antisense glinucletides Invlves synthesizing a strand f nucleic acid that will bind t mrna Deals with crrecting splicing in the DMB gene
Muscular Dystrphy Knwns: Becker and Duchenne MD are caused by a mutatin in the DMB gene Lack f specific prtein dystrphin Unknwns: Levels f dystrphin necessary t deter MD symptms Hw t maintain synthetic system
THE END-Questins?