Cystic Fibrosis Summary

To correct the mutation in the gene that lead to the development of cystic fibrosis, a team of Yale researchers led by Dr. Peter Glazer (Chairman, therapeutic radiology), Mark Saltzman (Chairman, biomedical engineering), and Dr. Marie Egan (professor of paediatrics and of cellular and molecular physiology), the collaborative team operated certainartificial molecules identical to DNA known as Peptide Nucleic Acids (PNAs), as well as donor DNA, to recover the genetic abnormality.Professor Egan explained that the PNAare synthetic oligonucleotide analogue, repellent of protease and nuclease degradation that clamps to the DNA close to the mutated region and stimulating DNA repair and recombination pathways in cells upon sequence-specific triplex formation. These triplex-forming PNA molecules along with donor DNA in certain biodegradable nanoparticles are used to repair F508del, the cause of Cystic Fibrosis. …show more content…
To shieldthe PNA molecules for gene editing action, PLGA nanoparticles were stuffed with PNAs and donor DNAs with the help of double emulsion solvent evaporation technique. Nanoparticles assisting the donor DNAs and the various PNA molecules were then tested on CF bronchial epithelial (CFBE) cells containing