Molecular Therapy Nucleic Acids

Scope

Molecular Therapy — Nucleic Acids is an international, open-access journal publishing top-quality basic, translational, and clinical research in the broad fields of nucleic acid-based therapeutics to treat and/or correct genetic and acquired disease. Subject areas include but are not limited to: development of therapeutics based on nucleic acids and their derivatives, vector development and design for the delivery of RNA-based therapeutics, applications of gene-modifying agents including triplex-forming oligonucleotides and enzymes such as Zn finger nucleases, pre-clinical target validation, safety/efficacy studies, and clinical trials. Other key topics include: Applications of DNA-based oligonucleotides and their derivatives Gene vectors for delivery of RNA-based therapies Development of nanoparticles and other delivery agents siRNAs, shRNAs, and miRNAs Aptamers, ribozymes, and DNAzymes Peptide nucleic acids Gene-modifying approaches including enzymes, triplex-forming nucleic acids, and other approaches Development of new nucleic acid chemistries for altering gene expression Mechanisms of gene and nucleic acid transfer/transfection Vector trafficking and biodistribution.