Advances in the development and delivery of nucleic acid-based therapeutics.

Abstract

Recent advancements in experimental and computational methods along with growing gene sequencing libraries have enabled rapid development of nucleic acid-based therapeutic design for a wide variety of diseases. New drugs, delivery tools and methods have enabled precise and efficient therapy at a molecular scale. This review covers the current landscape and future directions of methods for gene and cell therapies, as well as their applications, advantages, and limitations. We present self-amplifying RNA, circular RNA, CRISPR systems, antisense oligonucleotides, and antibody-oligonucleotide conjugates, as well as CAR modification of T-cells, natural killer cells, and macrophages, with a brief discussion of modified stem cells. We also discuss developments in viral and non-viral delivery systems for their applications in cell and gene therapies.