Molecular Therapy - Nucleic Acids

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.