LIPID NANOPARTICLE FORMULATIONS COMPRISING NUCLEIC ACID MIMICS

作者: Ying Bo , Sim Derek

DOI:

关键词: NanoparticleNucleic acidBiochemistryIntracellularGenome editingChemistry

摘要: Described herein are lipid nanoparticles (LNPs), comprising neutral or positively charged nucleic acid mimics (NPNAMs) and optionally acids, compositions thereof. Also described methods of preparing LNPs NPNAMs, use for intracellular gene editing. In particular, the NPNAMs acids may be used in correction and/or treatment a genetic disorder, disease, condition subject.

参考文章(20)
Bob Dale Brown, Lipid delivery formulations ,(2012)
Ernest Ludwig Eliel, Samuel H. Wilen, Tables of resolving agents and optical resolutions ,(1972)
Jean Jacques, André Collet, Samuel H. Wilen, Enantiomers, Racemates, and Resolutions ,(1981)
Peter G. M. Wuts, Theodora W. Greene, Protective groups in organic synthesis ,(1981)
Jason P Schrum, Noubar B Afeyan, Gregory J Sieczkiewicz, Stephane Bancel, Antonin De Fougerolles, Sayda M Elbashir, Kenechi Ejebe, Engineered nucleic acids encoding a modified erythropoietin and their expression ,(2012)
Antonius Lobberding, Burkhard Mielke, Eckhard Schwenner, Christoph Schwemler, Udo Stropp, Thorsten Potter, Wolfgang Springer, Axel Kretschmer, Nucleic acid-binding oligomers possessing C-branching for therapy and diagnostics ,(1994)
Michael Egholm, Peter E. Nielsen, Rolf H. Berg, Ole Buchardt, Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility ,(1997)
Irvin D. Kuntz, Vibhakar J. Shah, George L. Kenyon, Peptide-based nucleic acid mimics(PENAMS) ,(1995)