GeneTherapy

GeneTherapy Springer Nature's monthly journal publishing high-quality research and insights in gene and stem cel

Gene Therapy covers both the research and clinical applications of the new genetic therapy techniques currently being developed. Over the last decade, gene therapy protocols have entered clinical trials in increasing numbers and as they cover a wide spectrum of diseases, these studies promise to unite the diverse organ-based specialties into which modern medicine has become divided. Gene Therapy c

overs all aspects of gene therapy as applied to human disease, including:

- Novel technological developments for gene transfer, control and silencing
- Basic science studies of mechanisms of gene transfer and control of expression
- Pre-clinical animal model systems and validation studies
- Clinical trial reports which have significant impact for the field
- Gene-based vaccine development and applications
- Cell-based therapies including all aspects of stem cells and genetically modified cellular approaches

Gene doping can hide in plain sight. This study shows codon changes let transgenes evade qPCR -yet sequencing still flag...
14/11/2025

Gene doping can hide in plain sight. This study shows codon changes let transgenes evade qPCR -yet sequencing still flags them. Implications for anti-doping and gene therapy monitoring.
📖https://doi.org/10.1038/s41434-025-00569-y

A minimally invasive ultrasound-guided AAV9 delivery in utero shows safe, widespread gene transfer—no toxicity, minimal ...
13/11/2025

A minimally invasive ultrasound-guided AAV9 delivery in utero shows safe, widespread gene transfer—no toxicity, minimal immune response, strong translation potential for severe monogenic and mitochondrial diseases.
📖https://doi.org/10.1038/s41434-025-00551-8

Gene therapy doesn’t end at infusion. This review of 103 long-term follow-up studies on ClinicalTrials.gov shows wide va...
12/11/2025

Gene therapy doesn’t end at infusion. This review of 103 long-term follow-up studies on ClinicalTrials.gov shows wide variation in safety monitoring and adverse-event terms—making comparisons difficult.
📖https://doi.org/10.1038/s41434-025-00571-4

Focused ultrasound intrathecal delivery of  -SaCas9 boosts brain editing: ~2000× more hippocampal Cas9 and fewer pathoge...
10/10/2025

Focused ultrasound intrathecal delivery of -SaCas9 boosts brain editing: ~2000× more hippocampal Cas9 and fewer pathogenic glycogen inclusions with lower neuroinflammation in a mdoel
🔗 https://www.nature.com/articles/s41434-025-00517-w

Commentary: Aortic aneurysm risk is high, but many AAVs favor liver. Proposed roadmap:   RFTEKPA, liver-detargeting edit...
09/10/2025

Commentary: Aortic aneurysm risk is high, but many AAVs favor liver.
Proposed roadmap: RFTEKPA, liver-detargeting edits, vascular smooth muscle cell promoters and miR-122 “off” switches for sharper, safer aortic delivery.
🔗 https://www.nature.com/articles/s41434-025-00526-9

A new  -of-care   test detects AAV8-binding antibodies in plasma, serum, and blood in under 20 minutes aligns with lab-b...
08/10/2025

A new -of-care test detects AAV8-binding antibodies in plasma, serum, and blood in under 20 minutes aligns with lab-based ELISA and NAb assays. Assisting rapid identification of patients that can safely receive .
🔗 https://www.nature.com/articles/s41434-025-00559-0

New study finds mutations in HSV-1 based replication-defective vectors can induce syncytial variants, causing hyperexcit...
02/10/2025

New study finds mutations in HSV-1 based replication-defective vectors can induce syncytial variants, causing hyperexcitable neurons. Critical insight for CNS safety.
📖 nature.com/articles/s41434-025-00566-1

Pre-existing immunity remains a critical barrier in AAV  . A dual-assay approach -combining total antibody and transduct...
01/10/2025

Pre-existing immunity remains a critical barrier in AAV . A dual-assay approach -combining total antibody and transduction inhibition offers a practical way to better stratify patients for therapies.
📖:https://www.nature.com/articles/gt201795

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