Precision Cellular Reprogramming for Scalable and Consistent Human Neurodegenerative Disease Models
CRISPR/Cas9, a ground-breaking tool for altering genes with the potential to fully cure genetic diseases, has made its way into the clinic, accelerating research on new therapies using this technology. Since cell networks are amazingly complex and still not fully understood, modifying a single gene to treat a patient can have various effects that need to be determined. Answering the questions of what is happening, and to which extend is it happening requires an untargeted and very sensitive approach.
Here, experimental design, depth of analysis, target coverage and statistical challenges will be discussed to help biopharmaceutical drug developers ensure safe and efficient gene therapy products.
During this talk we discuss:
- Experimental design and analysis
- The benefits of using state-of-the-art SWATH DIA