Meeting+Minutes

Wednesday 11/30 Sharmila - background Lillian - SMA and muscular dystrophy morpholino more stable than omethyl, ptc, omethyl -- ferylonopathy, sma, collagen, separate FDA for each exon, abi/avi, european intramuscular but now iv unpublished but worked! 60% covered if remove exons 44-57 studies on 52? large gene hope for being active, 27% recovery effective cure, very encouraging, 20-25 bp micro rna, no off target effects, no side effects - technology done in duchenne, delivery done by isis - nanoliposomes

Lauren Yannis

Wednesday 11/9/2011 LOXIN -figure out what's wrong with statins -IP -raising funds, budget -CEO, management -logo, name -technology -indication, further indications -clinical trial plan -FDA -issues and how to fix it

Friday, September, 23 Brief Notes (among the ideas we discussed)
 * Nanocapsules that cross the blood-brain barrier. Applications for delivery of neuro drugs.
 * Gene knockdown that causes very low HDL, LDL, triglycerides à negligible risk of heart attack and stroke
 * PUF proteins can be engineered to bind very specifically to mRNA sequences. “enabling selective binding of a specific RNA target to investigate or manipulate any aspect of its metabolism." (Both activating and repressing PUF proteins have been discovered). A protein-mediated method of gene knock-down or editing.
 * Genome modification with Zinc Finger Nucleases or TAL Effectors.
 * Protozoa-mediated protein and drug delivery system.
 * Methods for assay including electroporation.
 * Nano-scale lasers as a possibility of targeted therapy.
 * Bacteria which home to tumors and could potentially deliver proteins.
 * Artificial chromosomes.
 * Alternate-splicing technology with small RNAs to bind to splice sites à skipping of problematic exons for splicing or mutation disorders / skipping of functional exons to cause deletion.

Notes from 9.20.1990 - Yes, we've been working on this project since we were born. ideas: -using splicing to fix gain of function diseases -splice out STOP codon in exon for disease other than Duchenne -use nanocapsules to deliver drugs to brain

Moving forward: - Expand idea base for meeting on Friday