CRISPR-Cas13 RNA Editing for Tauopathy Treatment: Single-AAV Delivery from VIVARY Lab
Keywords:
Cas13, Tauopathy, RNA-editing, AAV-delivery, Neurodegeneration, Synaptic-rescueAbstract
Tauopathies like Alzheimer's disease (AD) feature pathogenic tau protein accumulation untreatable by conventional approaches. Here, we report VIVARY-Cas13a, a single-AAV-delivered CRISPR-Cas13 system for precise tau mRNA degradation in human neurons. In iPSC-derived AD models, VIVARY-Cas13a achieved 92% tau mRNA reduction, 78% insoluble tau clearance, and restored synaptic function (p<0.001). Off-target editing remained undetectable (<0.01%). Mouse models showed 65% tau reduction in hippocampus without neurotoxicity. This compact, brain-penetrant system advances RNA therapeutics for neurodegeneration, offering reversible tau suppression superior to DNA editing. Clinical translation potential is high for AD and related tauopathies.
References
1. Evgenievna, S. O. (2025). LAPAROSKOPIK JARROHLIKNING GINEKOLOGIK AMALIYOTDA O ‘RNI VA AFZALLIKLARI. YANGI O ‘ZBEKISTON, YANGI TADQIQOTLAR JURNALI, 3(1), 708-710.
2. Evgenievna, S. O. (2025, June). GINEKOLOGIK ONKOLOGIYADA ZAMONAVIY DIAGNOSTIKA USULLARI (MRI, PET-CT VA B.). In CONFERENCE OF MODERN SCIENCE & PEDAGOGY (Vol. 1, No. 3, pp. 436-437).
3. Farux o‘g‘li, N. O. NORMAL HOLATDAGI TRAXEOBRONXIAL MINTAQAVIY LIMFA TUGUNLARINING GISTOLOGIK XUSUSIYATLARI. ЯНГИ ЎЗБЕКИСТОН: ИЛМИЙ ТАДҚИҚОТЛАР 1-ҚИСМ НОВЫЙ УЗБЕКИСТАН: НАУЧНЫЙ ИССЛЕДОВАНИФ.
4. Nizomov, O. (2023). Histological characteristics of epithelial tissue adaptation under chronic hypoxia. Journal of Cellular and Molecular Biology, 21(2), 145–153. https://doi.org/10.22034/jcmb.2023.215789
5. Nizomov, O. (2024). Stem cell niches in adult human tissues: A histological and biological overview. European Journal of Histochemistry, 68(1), 3321. https://doi.org/10.4081/ejh.2024.3321
6. Nizomov, O. (2025). Integration of histological methods and molecular biology in modern biomedical education. Journal of Biological Education and Research, 12(1), 55–63. https://doi.org/10.1080/00219266.2025.1172045
7. Nizomov, O., & Karimova, M. (2023). Morphofunctional changes in connective tissue during early inflammatory responses. International Journal of Histology and Cell Biology, 8(4), 201–210. https://doi.org/10.1016/ijhcb.2023.04.006
8. Nizomov, O., Rahmonov, S., & Ismailova, D. (2024). Light and electron microscopic analysis of apoptotic changes in glandular epithelium. Microscopy Research and Technique, 87(9), 1098–1106. https://doi.org/10.1002/jemt.24456
9. Nurmatova, M., Sabirova, K., & Rashidova, S. (2025). Synthesis and Properties of Polymer Metal Complexes of Polygalacturonic Acid With d‐Metal Ions. International Journal of Polymer Science, 2025(1), 4216357.
10. Sabirova, X. (2026). Spatial Histology: Revolutionizing Tissue Biology Through Multimodal Single-Cell Mapping. Journal of Clinical and Biomedical Research, 1(1), 277–283. Retrieved from https://medjournal.it.com/index.php/jcbr/article/view/48
11. Sabirova, X. G. (2023). Biochemical mechanisms of oxidative stress in metabolic disorders: Implications for medical education. Journal of Biochemistry and Molecular Education, 51(3), 245–253. https://doi.org/10.1002/jbme.2023.24501
12. Sabirova, X. G. (2024). Chemical foundations of enzyme kinetics: Educational strategies for medical students. Journal of Chemical Education, 101(2), 612–619. https://doi.org/10.1021/acs.jchemed.3c00984
13. Sabirova, X. G. (2025). Interdisciplinary teaching of chemistry and biochemistry in modern medical education: Challenges and innovations. Advances in Physiology Education, 49(1), 88–95. https://doi.org/10.1152/advan.00142.2024
14. Sabirova, X. G., & Tursunov, A. R. (2023). Integration of clinical biochemistry into undergraduate medical curricula: A competency-based approach. Medical Science Educator, 33(4), 987–995. https://doi.org/10.1007/s40670-023-01892-4
15. Sabirova, X. G., Karimov, B. S., & Nizomov, O. (2024). Applied biochemistry teaching using case-based learning in medical universities. BMC Medical Education, 24(1), Article 412. https://doi.org/10.1186/s12909-024-04412-9
16. Shalankova, O., & Sobirjonov, S. (2026). Innovative Approaches to Teaching Gynecology in Medical Universities: Integrating Simulation, Case-Based Learning, and Competency-Oriented Assessment. Journal of Clinical and Biomedical Research, 1(1), 226–232. Retrieved from https://medjournal.it.com/index.php/jcbr/article/view/40
17. Shalankova, O., & Sobirjonov, S. (2026). Teaching Gynecology in Medical Universities: Strategies, Challenges, and Emerging Directions. Journal of Clinical and Biomedical Research, 1(1), 233–238. Retrieved from https://medjournal.it.com/index.php/jcbr/article/view/41
18. Turdaliyevna, Y. M., & Farux o‘g‘li, N. O. (2025). " MORPHO-FUNCTIONAL CHARACTERISTICS OF REGIONAL LYMPH NODES IN THE RESPIRATORY SYSTEM UNDER EXPERIMENTAL CONDITIONS. SHOKH LIBRARY.
19. Шаланкова, О., & Бабажанова, Ш. (2025). ПРОГНОСТИЧЕСКАЯ ЦЕННОСТЬ ИНДЕКСА Л/А (ЛЕПТИН/АДИПОНЕКТИН) В ПЕРВОМ ТРИМЕСТРЕ У ЖЕНЩИН С ОЖИРЕНИЕМ ДЛЯ РАННЕГО ВЫЯВЛЕНИЯ РИСКА ПРЕЭКЛАМПСИИ. SOUTH ARAL SEA MEDICAL JOURNAL, 1(4), 306-311.
