Commercially available isobaric tags employ N-hydroxy-succinimide (NHS) chemistry to target α- and ε-amino groups.
Here we applied the SpAAC chemistry to target specific azido-CCR5 variants with a peptide epitope-tagged cyclooctyne to bypass the known disadvantages in the Staudinger ligation such as insufficient reaction stoichiometry with azF and phosphine oxidation.
We describe here the methodical, cooperative comparison, in vitro (in DMD cells) and in vivo (in a transgenic mouse expressing human dystrophin), of 24 AOs of the phosphorodiamidate morpholino oligomer (PMO) chemistry designed to target exon 53 of the DMD gene, skipping of which could be potentially applicable to 8% of patients.
In this regard, our medicinal chemistry approach was to target both β1 and β2 subunits to activate all the AMPK heterotrimers that are present across multiple tissues.
Nevertheless, the prospect of possible siRNA applications, but also of promising applications in modern bioconjugation chemistry, prompted us to target 2′-azido 2′-azido RNA
Investigators in the Program are carrying out systematic genomic characterization of lung cancers to identify therapeutic targets, employing structural biology and medicinal chemistry to effectively target the genomic changes, and defining mechanisms of resistance to develop therapeutic strategies to overcome the acquired resistance.
He was also co-founder of Calculario, a materials discovery company that leverages quantum chemistry and machine learning to target advanced materials in a range of high-value markets.
The well-established surface chemistry for GNPs allows one to target cells specifically by conjugating different moieties (e.g., antibodies, peptides, and DNAs) to the nanoparticles [6], and the conjugates have good biocompatibility and maintain their original biological activity after conjugation.
With variations in the size, coating thickness, surface chemistry, and targeting ligands, these nanoparticle probes can be tailored to target specific organs, cells, or even molecular markers of different diseases in vivo.
