Nanoparticles that deliver triplex-forming peptide nucleic acid molecules correct F508del CFTR in airway epithelium

Autor:	Kavitha Anandalingam, Peter M. Glazer, Rachel J. Fields, Lee A. Polikoff, W. Mark Saltzman, Elias Quijano, Marie E. Egan, John P. Geibel, Anisha Gupta, Yong Kong, Raman Bahal, Nicole Ali McNeer, Sascha Kopic, Christina Caputo
Rok vydání:	2015
Předmět:	Genetic enhancement General Physics and Astronomy Biology medicine.disease_cause Cystic fibrosis General Biochemistry Genetics and Molecular Biology 03 medical and health sciences chemistry.chemical_compound 0302 clinical medicine Genome editing medicine 030304 developmental biology 0303 health sciences Mutation Multidisciplinary Peptide nucleic acid General Chemistry medicine.disease Cystic fibrosis transmembrane conductance regulator 3. Good health chemistry Biochemistry 030220 oncology & carcinogenesis biology.protein Respiratory epithelium DNA
Zdroj:	Nature Communications. 6
ISSN:	2041-1723
Popis:	Cystic fibrosis is a lethal genetic disorder commonly caused by the F508del mutation which is not amenable to gene therapy. Here, the authors use triplex-forming PNA molecules and donor DNA in biodegradable polymer nanoparticles to correct F508del and achieve clinically relevant levels of gene editing.
Databáze:	OpenAIRE
Externí odkaz:	https://explore.openaire.eu/search/publication?articleId=doi_________::cd01d4564746145641ff568a40bd78bd https://doi.org/10.1038/ncomms7952 Zobrazit plný text záznamu