Publications by authors named "Devam A Desai"
Front Pharmacol
September 2024
Article Synopsis
- In-vivo CRISPR Cas genome editing therapy involves the use of lipid nanoparticles, mRNA, and single guide RNA, requiring prediction models due to limited data on their distribution in the body.
- A Quantitative Systems Pharmacology (QSP) model was developed to assess the pharmacokinetics and pharmacodynamics of CRISPR therapies, based on studies involving transthyretin amyloidosis and LDL-cholesterol reduction in mice and non-human primates transitioning to humans.
- The QSP model successfully described the biodistribution, key mechanisms post-injection, and the effect on serum TTR levels, demonstrating its potential in advancing the development of CRISPR-based therapies.
