Therapeutics based on nucleic acid systems act through diverse mechanisms, offering numerous advantages and potential therapeutic benefits due to their molecular properties. These include the identification, production, and delivery of human nucleic acids (DNA and RNA) into cells and tissues of different organs. These genetic materials can modify cellular activity by modulating gene activity or regulating expression levels. To do this, highly specific changes can be performed, such as the insertion, substitution, and deletion of sequences in nucleic acids to edit a gene or correct an inherited mutation. Gene therapy, gene-modified cell therapy, and genome editing are examples of these applications. Viral systems involving adenoassociated virus, herpes simplex virus-1, and lentivirus, as well as non-viral nucleic acid systems such as anti-sense oligonucleotides, small RNAs (microRNAS and small interfering RNAs), and aptamers, have been tested. Recent clinical trials of nucleic acid-based therapies have shown remarkable safety and therapeutic benefits. Some of these are currently approved drugs for the treatment of Duchenne muscular dystrophy, spinal muscular atrophy, dystrophic epidermolysis bullosa, haemophilia A, sickle-cell disease, and other diseases. This chapter will describe the nucleic acid-based systems for regenerative medicine and the state-of-the-art strategies for nucleic acid-based systems.