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journal.txt
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journal.txt
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# Large-pharma
The Large Pharmaceutical Company Perspective
```{r}
cptid <- "2017-04-07-skyoon-largepharma"
DOI <- "10.1056/NEJMra1510069"
Journal(cptid, DOI)
```
http://www.nejm.org/doi/pdf/10.1056/NEJMra1510069
- Rotavirus Efficacy and Safety Trial (REST)
- Phase 3 trial
- 70000 infants and children (RotaTeq) -
- Previously reported s/e of rotavirus vaccine (RotaShiled)
- 2006 approved.
- Intussusception
- 1/100,000 - Intu (RotaTeq) vs 1 to 3 of 10,000 (RotaShiled)
- Alendronate
- Fosamax by Merck, bisphosphonate
- Clinical Trial based on monkey experiments
- Fx Intervention Trial (FIT)
# Genome Editing Techniques and Their Therapeutic Application
```{r}
cptid <- "2017-04-07-yscho-genomeEdit"
DOI <- "10.1002/cpt.542"
Journal(cptid, DOI)
```
- Potential of a DSB (Double-Strand Break)
- CRISPR/Cas9
- Argonaute
- Non-homologous end joining (Frequency 10-50%)
- Finding the most promising therapeutic application - desired genetic outcome, cell type, delivery method (Difficult!)
- HSV-1 (Cornea) CAR-T (Destroying TCR and CD52)
- Power of Gene Addition
- Efficiency vs precision
- DSB vs Retrovirus vs Transposon
- Retro/Transposon - lack DNA sequence spec
- site-specific recombinases - all-in-one enzyme
- cre resolvase
- cas9