Gene Therapy

Latest research

1. Cost-effectiveness analysis of vagus nerve stimulation in drug-resistant epilepsy

   • Weixi Xiong
   • Lu Lu
   • Dong Zhou

   in Acta Epileptologica

   Article Open access 01 November 2025

2. CRISPR-based gene therapy for huntington’s disease: current advances and future prospects

   • Amna Zaheer
   • Noha Mohamed AboQuella
   • Syed Ijlal Ahmed

   in Neurogenetics

   Article 31 October 2025

   

3. Upregulation of CCK3R leading to the suppression of chronic seizures

   • Ling He
   • Liyang Zhang
   • Jufang He

   in Translational Psychiatry

   Article Open access 31 October 2025

   

4. Personalized gene editing helped one baby: can it be rolled out widely?

   in Nature

   Article 31 October 2025

   

5. Gene Therapy for Inherited Cardiac Arrhythmias

   • Mara R. Turkieltaub Paredes
   • Mohit Hulsurkar
   • Xander H. T. Wehrens

   in Journal of Cardiovascular Translational Research

   Article 30 October 2025

   

6. A targeted vector for brain endothelial cell gene delivery and cerebrovascular malformation modelling

   • Jun-Liszt Li
   • Zhanying Bi
   • Woo-ping Ge

   in Nature Biomedical Engineering

   Article Open access 29 October 2025

   

7. Cardiac troponin T promoters lead the way: optimizing cardiac specificity in AAV-mediated gene therapy

   • Anagh Astavans
   • Andreas S. Barth

   in Gene Therapy

   Article 29 October 2025

8. Therapeutic precision gene editing of cholesterol pathways as a gene therapy strategy for cardiovascular disease

   • Ezgi Erbasan
   • Melike Aliciaslan
   • Salih Sanlioglu

   in Gene Therapy

   Article 28 October 2025

   

9. Can Gene Therapy Transform the Treatment Landscape of Posterior Segment Eye Diseases? A Comprehensive Review of Recent Advancements

   • Kai-Yang Chen
   • Hoi-Chun Chan
   • Chi-Ming Chan

   in Drugs

   Article Open access 25 October 2025

   

10. Genome-edited allogeneic CAR-T cells: the next generation of cancer immunotherapies

    • Jingchao Su
    • Yifei Zeng
    • Sanfang Tu

    in Journal of Hematology & Oncology

    Article Open access 24 October 2025

    

11. Trends in gene therapy delivery technologies

    • Emily Capra
    • Ling Liu
    • Raman Thadani

    in Nature Reviews Drug Discovery

    Article 23 October 2025

    

12. A HO-1 gene knockout using a NanoCRISPR scaffold suppresses metastasis in mouse models

    • Ning Wang
    • Zichao Luo
    • Changyang Gong

    in Nature Biomedical Engineering

    Article 22 October 2025

    

13. Chimeric antigen receptor modified hematopoietic stem cells (CAR-HSCs) arm all immune forces for anti-tumor in mice

    • Tao Wang
    • Ping Liu
    • Jianmin Yang

    in Experimental Hematology & Oncology

    Article Open access 22 October 2025

    

14. Characteristics of long-term follow-up studies for gene therapies registered on ClinicalTrials.gov

    • Carolyn Riley Chapman
    • Ava Glazier
    • Barbara E. Bierer

    in Gene Therapy

    Article 22 October 2025

    

15. Cardiac gene therapy makes a comeback

    • Oliver J. Müller
    • Susanne Hille
    • Anca Kliesow Remes

    in Nature Medicine

    Article 21 October 2025

    

16. Assessment of AAV/polyketal chimeric nanoparticles for ocular gene transduction using an animal model

    • Brenda Fernanda Moreira Castro
    • Margareth Lugin
    • Silvia Ligório Fialho

    in Drug Delivery and Translational Research

    Article 21 October 2025

    

17. Cardiotropic AAV gene therapy for heart failure: a phase 1 trial

    • Timothy D. Henry
    • Eugene S. Chung
    • Roger J. Hajjar

    in Nature Medicine

    Article Open access 21 October 2025

    

18. Sickle Cell Disease and Gene Therapy Among African Americans: A Dilemma and Challenge

    • Elizabeth Armstrong-Mensah
    • Ato Kwamena Tetteh
    • David Armstrong-Mensah

    in Journal of Racial and Ethnic Health Disparities

    Article 20 October 2025

    

19. Muscle-specific gene editing therapy via mammalian fusogen-directed virus-like particles

    • Shi-Kun Zhou
    • Jing-Tong Luo
    • Jun Wang

    in Nature Communications

    Article Open access 15 October 2025

    

20. Engineering a streamlined virus-like particle for programmable tissue-specific gene delivery

    • Jiabao Han
    • Hanyu Bai
    • Wei Li

    in Nature Communications

    Article Open access 15 October 2025

    

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