Yannis


 * For Friday 9.23**

__Caltech Tech Transfer office__
 * Method for targeted deliver of vaccine antigens to DCs using lentiviruses [[file:Antigen delivery to DCs using lentivirus.pdf]]
 * Method for incorporating non-natural aminoacids into proteins in order to label and follow them
 * Not sure how it is applicable but I thought it was cool
 * Synthetic RNA device for modulating gene expression [[file:PNAS synthetic RNA system.pdf]]

Potential applications: Disorders arising from mis-splicing
 * Assisted Splicing:** Using engineered protein-RNA complexes to target mRNA and enhance splicing




 * Assisted exon skipping**: Researchers used an oligonucleotide complementary to the sequences that direct splicing of dystrophin in patients with Duchenne muscular dystrophy. In those patients, a mutation in a particular exon was causing dystrophin translation to stop early and resulted in a dysfunctional dystrophin. The oligo bound to the sequences and prevented the inclusion of that exon in the RNA, thus restoring the functional protein. Can we apply this to other disorders caused by early STOP codons?


 * In Bauman et al. (2010) shown that splice switching oligonucleotides (SSOs) can be used to switch from a cancer inducing variant to an apoptotic variant in tumors in vivo, when SSOs were delivered using nanosomes. [[file:Splice Switching for cancer.pdf]]
 * The switch of CD44 splicing causes EMT in breast cancer. [[file:CD44 splice switch for breast cancer metastasis.pdf]]

Potential Applications: Fine-tuning protein expression to reach a very specific level
 * Promoter Engineering:** The investigators observed that commonly used promoters in the cells are rich in CpG dinucleotides. They proceeded to generate random sequences that fitted the features of known promoters but had no homology to real promoters. They show that the number of GC nuclelotides in the promoter cna be used to fine-tune expression levels in a dose dependent manner.

Potential applications: Diseases involving mitochondrial defects Question: Can you get cancer from mitochondrial gene mutations? If not, this would be a great way to sell gene therapy again!
 * Mitochondrial DNA gene therapy:** Altering the mitochondrial genome

PMID: 21918550 (Sorry, couldn't download the paper!)


 * Mitochondrial RNAi:** Exploiting tRNA sub cellular localization systems to target RNAi to the mitochondria. Could we use the same technique to deliver genes that we want to have expressed in the mitochondria?

Potential Applications: Loss Of Function disorders
 * Artificial Transcription factors:** Using zinc finger technology to engineer transcription factors at will: