BIOL 311 1st Edition Lecture 2 Current Lecture(Continued from Personalized Medicine)- Orphan diseasesDefined: <200,000 of the rarest diseases in the USAOrphan Act of 1983: Get patent for curing orphan disease1. FOP- Muscles/tendons turn into bones, fetal gene stays. The cause does not equal the cure (just because we know what causes this does not mean we know how to cure it)2. Pompe’s Disease- lys disease, myozome replacement enzyme therapy- Biotechnology ~Infinity Pharmaceuticals- If sheep eat corn lilies, offspring will have one eye  active ingredient is cyclopomine ($22,000/gram) – Synth anaology – IPI-P26 ~Digitalis  medieval times – William Withering (botanist) cured by Fox glove, a plant in which the active ingredient was digitalis- Religion/Bioethics~Human Embryonic Stem Cells (HESC)1. Human embryo origination (in a human blastocyst the cells from that inner masscan differentiate into any type of cell) as of 2010, clinical trials2. SCNT (Somatic Cell Nuclear Transfer) the nucleus of an egg its transferred into another cell and a fiberblast is inserted inside the egg3. STAP (Stimulus Triggered Acquisition or pluripotency) No embryos! The nucleus of a cell is put on a petri dish for ~30 minutes at a lower pH and it can then be used as any type of cell- Government~FDA  regulatory agency~NIH (national institute of health), NSF (National science foundation), DoD (Department of defense)  $$$$- Is human cloning possible???  Stem Cell TherapyImmortality?~Cells? Yes  Telomeres serve as a mitotic clock on normal cells, they shorten after eachcell division but through genetic manipulation telomeres may never shorten~Humans? Stem Cell Theory of Aging: ~30/40 years the number of stem cells decrease, less robust- Cell Biology and Bioterrorism ~Botnulinum Toxin (BoTox)  1g could kill 1 million people because it blocks the release of acetylcholine Legal with plastic surgery and cosmetics FDA RNAi (Interference RNA)  ds (double stranded) RNA that blocks a nativess (single stranded) RNA so that protein synthesis does not occur. siRNA is temporary while shRNA is permanent. Won a Nobel prize in 2006 Bad side: if it blocks the expression of a critical protein Good side: used as a drug to treat disease1. First clinical trial  wet macula degeneration  over secretion of VEGA (more blood vessels = angiogenesis) siRNA against VEGA (Currently in trial)2. Other applications: a) Huntington’s disease Product of over production of “bad” proteinb) p-glyco protein  multi drug resistant protein that goes onto cancer cells to help chemo be effective.3. Ricin-Protein-castorbean, mechanism of action: Binds to the ribosomes and activates them  no protein synthesis at all. Potent? One molecule can kill one cell- Special Topic: Stem Cells, Regenerative~Stem Cells: Replicate (divide) or differentiate~Sources of stem cells: embryos, adult tissue, fetal tissue – not useful, lab derived  iPSC  4 reprogrammed factors, can differentiate into any cell~ How potent are they? Totipotent – differentiates into any cell, pluripotent – differentiates into several cells, unipotent – can become one type of cell adult sources: baby teeth, cord blood, fat (liposuction), msc (mesosenchal stem cells) – stem cell therapy  News: Human embryo stem cells are very controversial  Human SCNT, clinical trials (stem cell therapy), STAP – fraud ??? regenerative medicine/tissue engineering~Regenerative medicine: Using our knowledge of cells to repair our bodies~Stem cells  best source for regenerative medicine and tissue engineering - Diseases that could be cured with stem cell therapy: Parkinson’s disease: slow death of dopamine secreting neurons Huntington’s disease: death of neuorons  Stroke: clinical trials Blindness  ocata  clinical trials with hESCs- BioLife and Bioheart  Stem cell Biology ~ BioHeart  cell therapy for the heart: taking cells from the body to bring them to the heart- Stem Cells and the Government: ~History (the government’s perspective):I. 2001  before: no federal money could be used for hESC researchII. 2001  George Bush said giving federal money would be okay if there are restrictions: pre-existing hESC lives ~64, no federal money used to create ew hESC lives.2009  Barack Obama said something similar to Bush’s 2001 policy: no federal money can be used to derive new hESC lives, increase number of government-regulated cell