Molecular Therapy Family of Journals

Assessing Germline Transmission Risk and First-in-Human Dose Selection for In Vivo Gene Therapies dlvr.​it/TWHBx7 #MTMCD

An integrated enzymatic and computational pipeline for quantifying off-target base-editing dlvr.​it/TWHBsW #MTMCD

Cas9 Immunogenicity and Translational Implications for CRISPR-Cas9 Mediated Gene Therapy Interventions dlvr.​it/TWGlVj #MTNA

A single-dose combinatorial gene therapy with AAV based FGF21 and SIRT1 impacts aging-associated metabolic and mitochondrial function dlvr.​it/TWGlMr #MTNA

A combination TLR7/8 and RIG-I agonist adjuvant reverts asthmatic allergic sensitization and prevents aggravated influenza infection in mice dlvr.​it/TWGVsr #MTMCD

A dual-function adenoviral system for transgene delivery and gene editing for host factor studies in a murine model of SARS-CoV-2 infection dlvr.​it/TWGVmF #MTMCD

Programmable Transcriptional Activation in Human iPSC-Derived Hepatocyte Metabolic Stress Culture for Target Discovery dlvr.​it/TWGNr7 #MTMCD

We're excited to share that Paloma Giangrande, PhD, has been appointed to a second term as Editor-in-Chief of Molecular Therapy Nucleic Acids (@MTJournalNews), beginning in January 2027. Congratulations on this well-deserved appointment! Read more: bit.ly/4iJGXHD

AAV6-Gsx1 promotes sustained functional recovery in a pilot long-term study after contusion spinal cord injury dlvr.​it/TWFz63 #MTMCD

Molecular engineering of zinc finger nucleases significantly enhances genome editing efficiency in cells dlvr.​it/TWFz1v #MTNA

In this issue dlvr.​it/TWFywk #genetherapy #celltherapy

Therapeutic Landscape of Folate Receptor-α Targeting: Platforms, Progress, and Clinical Translation dlvr.​it/TWFkyZ #genetherapy #celltherapy

Combination in vivo HSPC gene therapy for HIV by CCR5 knockout and decoy receptor dlvr.​it/TWF1hb #MTMCD

Surgical stress-induced hepatic regulatory T cells promote the development of colorectal liver metastases dlvr.​it/TWDrYL

Extracellular DNA amplifies autoimmunity in Aicardi-Goutières syndrome. dlvr.​it/TWDgfj #genetherapy #celltherapy

Development of an Efficient and Precise TadA-based C-to-G Base Editor Mi-zCGBE dlvr.​it/TWDgZj #genetherapy #celltherapy

Palmitoyl-protein thioesterase 1, PPT1: from a key enzyme driving the autophagy process to a promising target for drug development in cancer dlvr.​it/TWDZXH

Deep Phenotyping Reveals Evolution in T cell Phenotype after Hexaviral-specific T cell Infusion Post Hematopoietic Stem Cell Transplant dlvr.​it/TWDZVC #genetherapy #celltherapy

Preclinical efficacy and safety of a novel lentiviral vector for ex vivo gene therapy of p47phox-deficient chronic granulomatous disease. dlvr.​it/TWDZSP #genetherapy #celltherapy

Safe and efficacious gene therapy for sialidosis: From monocistronic to bidirectional AAV vectors encoding Neu1 and Ctsa dlvr.​it/TWDZP9 #MTMCD