Results 41 to 50 of about 326,770 (247)

Immunogenicity and toxicity of AAV gene therapy

Frontiers in Immunology, 2022
Gene transfer using adeno-associated viral (AAV) vectors has made tremendous progress in the last decade and has achieved cures of debilitating diseases such as hemophilia A and B.
Hildegund C. J. Ertl
doaj   +1 more source

Development of cell lines with increased susceptibility to diverse adeno-associated viral vectors to enable in vitro potency assays

Molecular Therapy: Methods & Clinical Development
Vectors based on adeno-associated viruses (AAVs) are promising therapeutic modalities used in gene therapy. Robust cell-based assays that demonstrate and quantify the potency of AAV vectors in expressing their transgene are needed for clinical ...
James Zengel, Emma S. Esterman, Anitha Ponnuswami, Nicholas R. Wall, Jan E. Carette   +4 more
doaj   +1 more source

Reducing the Risk of Adeno-Associated Virus (AAV) Vector Mobilization with AAV Type 5 Vectors [PDF]

, 2009
Current adeno-associated virus (AAV) gene therapy vectors package a transgene flanked by the terminal repeats (TRs) of AAV type 2 (AAV2). Although these vectors are replication deficient, wild-type (wt) AAV2 prevalent in the human population could lead ...
Li, C., Gray, S. J., Cockrell, S., Samulski, R. J., Hewitt, F. C., C. Li, S. J. Gray, Washburn, M., R. J. Samulski, M. Washburn, S. Cockrell, F. C. Hewitt   +11 more
core   +1 more source

CNS Transduction Benefits of AAV-PHP.eB over AAV9 Are Dependent on Administration Route and Mouse Strain

Molecular Therapy: Methods & Clinical Development, 2020
Adeno-associated viral (AAV) vectors are attractive tools for central nervous system (CNS) gene therapy because some vectors can cross the blood-brain barrier (BBB), allowing them to be used as minimally invasive treatments.
Sophie N. Mathiesen, Jasmine L. Lock, Lucia Schoderboeck, Wickliffe C. Abraham, Stephanie M. Hughes   +4 more
doaj   +1 more source

Redirecting AAV vectors to extrahepatic tissues

Molecular Therapy, 2023
Recombinant adeno-associated viral (AAV) vectors are the current benchmark for systemic delivery of gene therapies to multiple organs in vivo. Despite clinical successes, safe and effective gene delivery to extrahepatic tissues has proven challenging due to dose limiting toxicity arising from high liver uptake of AAV vectors.
Aravind Asokan, Shen Shen
openaire   +2 more sources

AAV vectors and titers.

, 2015
AAV vectors and titers.
Shannon E. Boye (248303), Herbert A. Reitsamer (183925), Seok Hong Min (407510), Zhonghong Zhang (751253), James J. Peterson (751252), William W. Hauswirth (53022), Barbara Bogner (751251), Sanford L. Boye (230301), Qing Ruan (349572)   +8 more
core   +1 more source

Rab1A Promotes Hepatic Steatosis by Suppressing Mitophagy via the Raf‐1/ERK1/2/PINK1 Signaling Axis

Advanced Science, EarlyView.
Why does fat accumulate in the liver? Our study reveals Rab1A as the molecular switch that silences the cell's mitochondrial cleanup crew. Turning Rab1A off reactivates mitophagy, clears excess fat, and reverses fatty liver disease in mice—offering a promising new therapeutic target for MASLD, a growing global health challenge.
Li Zhang, Jianhua Li, Huilu Zhang, Qi Qin, Yichen Huang, Xiaofan Tian, Chao Sun, Binbin Li, Zhengxin Wang, Xin Zhang   +9 more
wiley   +1 more source

Systemic gene transfer reveals distinctive muscle transduction profile of tyrosine mutant AAV-1, -6, and -9 in neonatal dogs

Molecular Therapy: Methods & Clinical Development, 2014
The muscular dystrophies are a group of devastating genetic disorders that affect both skeletal and cardiac muscle. An effective gene therapy for these diseases requires bodywide muscle delivery.
Chady H Hakim, Yongping Yue, Jin-Hong Shin, Regina R Williams, Keqing Zhang, Bruce F Smith, Dongsheng Duan   +6 more
doaj   +1 more source

Gonadotropin‐Inhibitory Hormone (GnIH) and Its Receptor GPR147 Mediates Acute Stress‐Induced Activation of the Locus Coeruleus‐SAM Axis

Advanced Science, EarlyView.
ABSTRACT Gonadotropin‐inhibitory hormone (GnIH, also known as RFamide‐related peptide‐3, RFRP‐3) is a hypothalamic neuropeptide that regulates vertebrate reproduction via its receptor GPR147. Although GnIH is known to mediate stress‐induced reproductive suppression, its role in broader stress responses remains unclear.
Jiani Zhang, Hongyu Zhu, Wenqi Wang, Bingqian Shen, Yuanyuan Xin, Xingxing Song, Xuanyi Lu, Lixuan Li, Siting Chen, Xiaochen Zhang, Jialing Zeng, Haodong Zheng, Jianlong Lao, Ke Peng, Yixian Wei, Jiaming Zheng, Chuanhuo Hu, Xiaoye Wang, Xun Li   +18 more
wiley   +1 more source

LARGE GENE DELIVERY TO THE RETINA BY MULTIPLE AAV VECTORS [PDF]

, 2019
Retinal gene therapy with adeno-associated viral (AAV) vectors is safe and effective yet it is limited by AAV cargo capacity of about 5 kb. AAV transfer capacity which is expanded by dual AAV up to 9 kb would still not suffice for treatment of inherited ...
TORNABENE, PATRIZIA
core   +1 more source