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. As an official journal of the American Society of Gene & Cell Therapy, Molecular Therapy Nucleic Acids builds upon the success of Molecular Therapy in publishing important peer-reviewed research, cutting-edge reviews, and commentaries targeted to advances in gene- and oligonucleotide-based therapies. Molecular Therapy Nucleic Acids's 2024 impact factor is 6.1

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, nucleic acid immunity, safety/efficacy studies, and clinical trials.

To accommodate the rapid growth of Molecular Therapy Nucleic Acids, starting in 2022, articles will be submitted and published in one of following subsections covering key topics.

Subsections include:

Bioinformatics

• AI

• NGS

• mathematical modeling

• phylogenetic oligo arrays

Clinical applications

• Original research featuring clinical or translational studies

• Case series or first-in-human trials with significant implications

• Early-phase clinical trial results (phase I/II)

• Advanced preclinical research with strong potential for clinical translation

Delivery strategies

• chemistry

• ligands

• lipid nanoparticles

• localized delivery applications

• polymeric nanoparticles

• systemic delivery

• targeted delivery

• viral vectors

Non-coding RNAs

• circRNAs

• lncRNAs

• miRNAs

• siRNAs

• regulatory guidelines

Nucleic acid immunity

• Nucleic acid sensing: mechanisms of innate immune recognition and signaling by DNA and RNA sensors, including TLRs, RIG I–like receptors, and cGAS.

• Pathway regulation: molecular control of activation, amplification, and resolution of nucleic acid–triggered innate immune signaling.

• Cell-autonomous antiviral immunity: antiviral restriction factors and nucleases involved in cell-autonomous antiviral immunity

• Nucleic acid homeostasis: innate immune sensing of nucleic acid homeostasis and nucleic acid metabolism

• Altered self nucleic acids: alterations of self nucleic acids (oxidation, displacement, modification, cleavage, damage) that activate or inhibit nucleic acid immunity

• Evolutionary antiviral systems: restriction-modification enzymes, CRISPR-Cas, cyclic-oligonucleotide-based antiphage signaling system, and RNAi in antiviral immunity

• Engineered nucleic acids: design, development, and enzymatic or chemical synthesis of nucleic acids that modulate innate immune receptor activity

• Nucleic acid therapeutics: strategies to prevent innate immune sensing in nucleic acid therapeutics (e.g., ASOs, small interfering RNAs (siRNAs), mRNAs, microRNAs (miRNAs), viral vectors)

• Therapeutic immune activation: therapeutic use of nucleic acids to induce innate immune activation (e.g., in cancer or infectious disease)

• Disease and aging: nucleic acid immunity and disease, including interferonopathies, lupus, neurodegeneration, and other aging-related diseases.

Oligonucleotides: diagnostics and biosensors

• aptamer-based biosensors

• molecular beacons

• nanostructured biosensors

• radiolabeled oligonucleotides

• rolling circle amplification-based biosensors

Oligonucleotides: therapies and applications

• clinical studies

• CMC

• development

• discovery

• preclinical assessment

• regulatory RNAs

RNA/DNA editing

• ADAR

• CRISPR

• HDR

• NHEJ

• TALEN

• ZFN