المؤلفون: Maccani, Andreas¹ (AUTHOR), Pachlinger, Robert¹ (AUTHOR), Eisenhut, Peter¹ (AUTHOR), Unterthurner, Sabine¹ (AUTHOR), Heider, Susanne¹ (AUTHOR), Repik, Christoph¹ (AUTHOR), Reuberger, Stefan¹ (AUTHOR), Andorfer, Peter¹ (AUTHOR), Lengler, Johannes¹ (AUTHOR), Kinastberger, Bernhard¹ (AUTHOR), Seczer, Dieter¹ (AUTHOR), Gruber, Petra¹ (AUTHOR), Apschner, Alexander¹ (AUTHOR) alexander.apschner@takeda.com, Micutkova, Lucia¹ (AUTHOR) lucia.micutkova@yahoo.com

المصدر: Journal of Biotechnology. Feb2025, Vol. 398, p127-132. 6p.

مصطلحات موضوعية: *GENE therapy, *ADENO-associated virus, *RECOMBINANT viruses, *MANUFACTURING processes, *PRODUCT quality, *GENE transfection

المؤلفون: Gioulvanidou, Maria¹ (AUTHOR), Sarklioglu, Selenay¹ (AUTHOR), Chen, Xinlei² (AUTHOR), Lebedeva, Irina V.² (AUTHOR), Inalman, Yeliz² (AUTHOR), Pohl, Mary Ann² (AUTHOR), Bourne, Lloyd² (AUTHOR), Andrew, David² (AUTHOR), Lorenz, Ivo C.² (AUTHOR), Stiles, Katie M.¹ (AUTHOR), Pagovich, Odelya E.¹ (AUTHOR), Hackett, Neil R.¹ (AUTHOR), Kaminsky, Stephen M.¹ (AUTHOR), de Mulder Rougvie, Miguel¹ (AUTHOR), Crystal, Ronald G.¹ (AUTHOR) geneticmedicine@med.cornell.edu

المصدر: Human Gene Therapy. Jan2025, Vol. 36 Issue 1/2, p11-27. 17p.

مصطلحات موضوعية: *ANTIBODY-dependent cell cytotoxicity, *ADENO-associated virus, *TRANSGENIC mice, *EOSINOPHILS, *INTRAVENOUS therapy, *MONOCLONAL antibodies

المؤلفون: Lan, Tian¹ (AUTHOR), Liang, Yongshun¹ (AUTHOR), Gan, Qingqiao¹ (AUTHOR), Liang, Hao¹ (AUTHOR) liangh@stu.gxmu.edu.cn

المصدر: Current Eye Research. Dec2024, p1-8. 8p. 4 Illustrations.

مصطلحات موضوعية: *ADENO-associated virus, *GLUTATHIONE peroxidase, *ULTRAVIOLET radiation, *SUPEROXIDE dismutase, *OXIDATIVE stress

المؤلفون: Li, Shuai¹ (AUTHOR), Wang, Xiaoyan¹ (AUTHOR), Lai, Kuan-Yu¹ (AUTHOR), Wert, Jonathan¹ (AUTHOR), Zhi, Li¹ (AUTHOR) li.zhi@regeneron.com, Shameem, Mohammed¹ (AUTHOR), Liu, Dingjiang¹ (AUTHOR) dingjiang.liu@regeneron.com

المصدر: Analytical & Bioanalytical Chemistry. Dec2024, Vol. 416 Issue 29, p7173-7182. 10p.

مصطلحات موضوعية: *ADENO-associated virus, *GENE therapy, *LEAKAGE, *CAPSIDS, *TRANSGENES, *SUCROSE

المؤلفون: Hassel, Keira R.^1,2,3 (AUTHOR), Gibson, Aaron M.¹ (AUTHOR), Šeflová, Jaroslava⁴ (AUTHOR), Cho, Ellen E.⁴ (AUTHOR), Blair, N. Scott¹ (AUTHOR), Van Raamsdonk, Catherine D.⁵ (AUTHOR), Anderson, Douglas M.⁶ (AUTHOR), Robia, Seth L.⁴ (AUTHOR), Makarewich, Catherine A.^1,7 (AUTHOR) cat.makarewich@cchmc.org

المصدر: Journal of Molecular & Cellular Cardiology. Dec2024, Vol. 197, p45-58. 14p.

مصطلحات موضوعية: *ANGIOTENSIN II, *ADENO-associated virus, *ENDOPLASMIC reticulum, *EIGENFUNCTIONS, *PHOSPHORYLATION

المؤلفون: Liu, Dequan¹ (AUTHOR), Li, Tian^1,2 (AUTHOR), Liu, Lei¹ (AUTHOR), Che, Xiangyu¹ (AUTHOR), Li, Xiaorui^1,3 (AUTHOR) xiaorui_0513@163.com, Liu, Chang^1,4 (AUTHOR) liuchang9011@aliyun.com, Wu, Guangzhen¹ (AUTHOR) wuguangzhen@firsthosp-dmu.com

المصدر: Cytokine & Growth Factor Reviews. Dec2024, Vol. 80, p109-120. 12p.

مصطلحات موضوعية: *GENE therapy, *ADENO-associated virus, *CENTRAL nervous system, *GENE targeting, *GENETIC disorders

المؤلفون: Chu, Simon N, Soupene, Eric, Sharma, Devesh, Sinha, Roshani, McCreary, Travis, Hernandez, Britney, Shen, Huifeng, Wienert, Beeke, Bowman, Chance, Yin, Han, Lesch, Benjamin J, Jia, Kun, Romero, Kathleen A, Kostamo, Zachary, Zhang, Yankai, Tran, Tammy, Cordero, Marco, Homma, Shota, Hampton, Jessica P, Gardner, James M, Conklin, Bruce R, MacKenzie, Tippi C, Sheehan, Vivien A, Porteus, Matthew H, Cromer, M Kyle

المصدر: Cell Reports. 44(1)

مصطلحات موضوعية: Biological Sciences, Hematology, Regenerative Medicine, Rare Diseases, Cooley's Anemia, Stem Cell Research - Nonembryonic - Human, Stem Cell Research - Nonembryonic - Non-Human, Transplantation, Genetics, Biotechnology, Sickle Cell Disease, Stem Cell Research, Blood, CP: Stem cell research, CRISPR, RNA sequencing, adeno-associated virus, genome editing, hematopoietic stem and progenitor cells, hemoglobin, red blood cells, α-thalassemia, Biochemistry and Cell Biology, Medical Physiology, Biological sciences

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/3p53m80j
https://escholarship.org/content/qt3p53m80j/qt3p53m80j.pdf

المؤلفون: Luthers, Christopher, Ha, Sung-Min, Mittelhauser, Annika, Morselli, Marco, Long, Joseph, Kuo, Caroline, Romero, Zulema, Kohn, Donald

المصدر: Molecular Therapy: Methods & Clinical Development. 32(4)

مصطلحات موضوعية: CD40LG, CRISPR-Cas9, HBB, adeno-associated virus 6, gene editing, hematopoietic stem cells, homologous recombination, non-homologous end joining, sickle cell disease

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/7245h38m
https://escholarship.org/content/qt7245h38m/qt7245h38m.pdf

المؤلفون: He, Hailun^1,2,3 (AUTHOR), Huang, Wenyue^1,2 (AUTHOR), Xiong, Lidan⁴ (AUTHOR), Ma, Cong^1,2,5 (AUTHOR), Wang, Yichong^1,2 (AUTHOR), Sun, Peihong^1,2 (AUTHOR), Shi, Dongxin^1,2 (AUTHOR), Li, Liangman⁶ (AUTHOR), Yan, Hongwei^1,2 (AUTHOR), Wu, Yan^1,2,7 (AUTHOR) jlwuyan@126.com

المصدر: Free Radical Biology & Medicine. Nov2024, Vol. 225, p630-640. 11p.

مصطلحات موضوعية: *MEMBRANE potential, *ADENO-associated virus, *LABORATORY mice, *CELL death, *MITOCHONDRIA

المؤلفون: Jieyi Ma^1,2, Siyi Zheng², Chenrui An³, Hui Han², Qiwen Li⁴, Ying Huang⁵, Gan Xiong², Shuang Chen², Siyao Guo², Zhaoyu Wang², Wei Wei², Yudan Shang³, Yushan Ji³, Cuiyun Yang⁵, Junho Choe^6,7,8,9, Quan Yuan⁴, Yong Fan³ yongfan011@gzhmu.edu.cn, Canfeng Zhang² zhangcf6@mail2.sysu.edu.cn, Shuibin Lin^2,10 linshb6@mail.sysu.edu.cn

المصدر: Proceedings of the National Academy of Sciences of the United States of America. 11/5/2024, Vol. 121 Issue 45, p1-12. 41p.

مصطلحات موضوعية: *PROTEOLYSIS, *ADENO-associated virus, *TRANSFER RNA, *GENETIC translation, *ENDOPLASMIC reticulum

المؤلفون: Karlin, David G.^1,2 (AUTHOR) davidgkarlin@gmail.com

المصدر: Viruses (1999-4915). Nov2024, Vol. 16 Issue 11, p1760. 11p.

مصطلحات موضوعية: *ADENO-associated virus, *LIFE cycles (Biology), *VIRAL proteins, *GENE therapy, *VIRAL replication

المؤلفون: Chen, Reed¹ reed.chen@duke.edu, Rey, Julian A.² julianrey1009@gmail.com, Tuna, Ibrahim S.³ tunais@radiology.ufl.edu, Tran, David D.⁴ david.tran@med.usc.edu, Sarntinoranont, Malisa⁵ msarnt@ufl.edu

المصدر: Journal of Biomechanical Engineering. Oct2024, Vol. 146 Issue 10, p1-10. 10p.

مصطلحات موضوعية: *FINITE element method, *ADENO-associated virus, *BRAIN tumors, *MAGNETIC resonance, *TARGETED drug delivery, *COMPUTATIONAL neuroscience

المؤلفون: Jacobsen, Kirsten Rosenmay¹ (AUTHOR) kirsten_rosenmay@yahoo.com, Mota, Javier² (AUTHOR) javier.mota@vrl.net, Salerno, Michelle³ (AUTHOR) msalerno@marshallbio.com, Willis, Alexis³ (AUTHOR) awillis@marshallbio.com, Pitts, Dennis² (AUTHOR) dennis.pitts@vrl.net, Denner, Joachim⁴ (AUTHOR) kirsten_rosenmay@yahoo.com

المصدر: Viruses (1999-4915). Oct2024, Vol. 16 Issue 10, p1613. 14p.

مصطلحات موضوعية: *GENE therapy, *ADENO-associated virus, *ANTIBODY titer, *ANIMAL experimentation, *XENOTRANSPLANTATION

مصطلحات جغرافية: GOTTINGEN (Germany)

المؤلفون: Yu, Shuangqing¹ (AUTHOR), Zhao, Qian² (AUTHOR), Zhang, Cengceng¹ (AUTHOR), Fu, Diyi² (AUTHOR), Zhu, Xueyang¹ (AUTHOR), Zhou, Jianfang¹ (AUTHOR), Ma, Wenhao¹ (AUTHOR), Dong, Zheyue³ (AUTHOR), Zhai, Xiaoliang⁴ (AUTHOR), Jiang, Lijie⁴ (AUTHOR), Han, Xiaohong² (AUTHOR), Zhang, Shuyang^2,5 (AUTHOR) shuyangzhang@pumch.cn, Wu, Xiaobing¹ (AUTHOR) shuyangzhang@pumch.cn, Dong, Xiaoyan¹ (AUTHOR)

المصدر: Viruses (1999-4915). Oct2024, Vol. 16 Issue 10, p1512. 14p.

مصطلحات موضوعية: *ADENO-associated virus, *GENE therapy, *QUALITY control, *SENSITIVITY & specificity (Statistics), *CLINICAL medicine

المؤلفون: Cuker, A.¹ adam.cuker@pennmedicine.upenn.edu, Kavakli, K.², Frenzel, L.³, Wang, J.-D.⁴, Astermark, J.⁵, Cerqueira, M. H.⁶, Iorio, A.⁷, Katsarou-Fasouli, O.⁸, Klamroth, R.⁹, Shapiro, A. D.¹⁰, Hermans, C.¹¹, Ishiguro, A.¹², Leavitt, A. D.¹³, Oldenburg, J. B.¹⁴, Ozelo, M. C.¹⁵, Teitel, J.¹⁶, Biondo, F.¹⁷, Fang, A.¹⁸, Fuiman, J.¹⁹, McKay, J.²⁰

المصدر: New England Journal of Medicine. 9/26/2024, Vol. 391 Issue 12, p1108-1118. 11p.

مصطلحات موضوعية: *BLOOD coagulation factor IX, *ADENO-associated virus, *GENE therapy, *HEMOPHILIA treatment, *BODY weight

المؤلفون: Zhou, Livia¹ (AUTHOR) kaizhou5757@126.com, Wang, Yafeng¹ (AUTHOR), Xu, Yiran² (AUTHOR), Zhang, Yaodong¹ (AUTHOR), Zhu, Changlian^2,3 (AUTHOR) changlian.zhu@neuro.gu.se

المصدر: Journal of Neuroinflammation. 9/19/2024, Vol. 21 Issue 1, p1-9. 9p.

مصطلحات موضوعية: *CENTRAL nervous system, *ADENO-associated virus, *NEURODEGENERATION, *THERAPEUTICS, *MICROGLIA

المؤلفون: Guan, Qiwen^1,2,3 (AUTHOR), Wang, Zhaojun^1,2 (AUTHOR), Zhang, Kai^1,2 (AUTHOR), Liu, Zhaoqian^1,2 (AUTHOR), Zhou, Honghao^1,2 (AUTHOR), Cao, Danfeng⁴ (AUTHOR), Mao, Xiaoyuan^1,2 (AUTHOR) xiaoyuanm@csu.edu.cn

المصدر: Journal of Advanced Research. Sep2024, Vol. 63, p73-90. 18p.

مصطلحات موضوعية: *TEMPORAL lobe epilepsy, *ADENO-associated virus, *BRAIN injuries, *KIDNEY physiology, *EPILEPSY, *PILOCARPINE

المؤلفون: Xiaoye Zheng¹, Ruixue Ma¹, Ershu He¹, Xin Peng¹, Wenhao Ma¹, Xueyan Zhang¹, Ying Li¹, Hanwei Li¹, Yanjiao Li¹ 754850549@qq.com, Zhiting Gong¹ gzhting@foxmail.com

المصدر: Alpha Psychiatry. Sep2024, Vol. 25 Issue 5, p641-647. 7p.

مصطلحات موضوعية: *DENTATE gyrus, *ADENO-associated virus, *SELF-expression, *MENTAL illness, *RECOMBINANT viruses

المؤلفون: van Lieshout, Laura¹ (AUTHOR) l.vanlieshout@oxb.com, Ota, Stacy¹ (AUTHOR), Adusei, Annie¹ (AUTHOR), Wiberg, Eli¹ (AUTHOR), Costa-Grant, Katrina¹ (AUTHOR), Lata, Dimpal¹ (AUTHOR), Dollive, Serena¹ (AUTHOR), Stanvick, Marissa¹ (AUTHOR), Iwuchukwu, Ifeyinwa¹ (AUTHOR), Golebiowski, Diane¹ (AUTHOR), Yin, Jin¹ (AUTHOR)

المصدر: Human Gene Therapy. Sep2024, Vol. 35 Issue 17/18, p767-776. 10p.

مصطلحات موضوعية: *ADENO-associated virus, *MESSENGER RNA, *CAPPING proteins, *VIRUS diseases, *ADENOVIRUSES

المؤلفون: Sia, Kian Chuan¹ (AUTHOR) sursiak@nus.edu.sg, Fu, Zhen Ying¹ (AUTHOR) surfz@nus.edu.sg, Mohd Rodhi, Siti Humairah¹ (AUTHOR) surshmr@nus.edu.sg, Yee, Joan Hua Yi² (AUTHOR) e0559166@u.nus.edu, Qu, Kun² (AUTHOR) kqu@nus.edu.sg, Gan, Shu Uin¹ (AUTHOR) surgsu@nus.edu.sg

المصدر: International Journal of Molecular Sciences. Aug2024, Vol. 25 Issue 15, p8342. 20p.

مصطلحات موضوعية: *ADENO-associated virus, *GENE therapy, *GENOME editing, *PLASMIDS, *CLINICAL medicine