Zobrazeno 1 - 10
of 591
pro vyhledávání: '"Guangping, Gao"'
Publikováno v:
Signal Transduction and Targeted Therapy, Vol 9, Iss 1, Pp 1-33 (2024)
Abstract Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene therapy owing to its minimal pathogenicity and ability to establish long-term gene expression in different tissues. Recombinant AAV (rAAV) has been engineer
Externí odkaz:
https://doaj.org/article/0d352d9c4b0c45b6a1cdc849a6677099
Autor:
Qing Xie, Xiupeng Chen, Hong Ma, Yunxiang Zhu, Yijie Ma, Leila Jalinous, Gerald F Cox, Fiona Weaver, Jun Yang, Zachary Kennedy, Alisha Gruntman, Ailing Du, Qin Su, Ran He, Phillip WL Tai, Guangping Gao, Jun Xie
Publikováno v:
EMBO Molecular Medicine, Vol 16, Iss 4, Pp 945-965 (2024)
Abstract Physiological regulation of transgene expression is a major challenge in gene therapy. Onasemnogene abeparvovec (Zolgensma®) is an approved adeno-associated virus (AAV) vector gene therapy for infants with spinal muscular atrophy (SMA), how
Externí odkaz:
https://doaj.org/article/4e7a21bfc0d24ec095ce22f8d8fa7575
Autor:
Nathan Bamidele, Han Zhang, Xiaolong Dong, Haoyang Cheng, Nicholas Gaston, Hailey Feinzig, Hanbing Cao, Karen Kelly, Jonathan K. Watts, Jun Xie, Guangping Gao, Erik J. Sontheimer
Publikováno v:
Nature Communications, Vol 15, Iss 1, Pp 1-13 (2024)
Abstract Nme2Cas9 has been established as a genome editing platform with compact size, high accuracy, and broad targeting range, including single-AAV-deliverable adenine base editors. Here, we engineer Nme2Cas9 to further increase the activity and ta
Externí odkaz:
https://doaj.org/article/df3d02ad296b4505893c699918456904
Publikováno v:
Neurotherapeutics, Vol 21, Iss 4, Pp e00391- (2024)
Adeno-associated virus (AAV)-based gene therapy is a clinical stage therapeutic modality for neurological disorders. A common genetic defect in myriad monogenic neurological disorders is nonsense mutations that account for about 11% of all human path
Externí odkaz:
https://doaj.org/article/df387ac4c6094baebea12d928cbd4626
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 2, Pp 102184- (2024)
Nonalcoholic steatohepatitis (NASH) represents a severe disease subtype of nonalcoholic fatty liver disease (NAFLD) that is thought to be highly associated with systemic metabolic abnormalities. It is characterized by a series of substantial liver da
Externí odkaz:
https://doaj.org/article/ced1dfbe0c184e66b1ecb302d9383bfd
Autor:
Hao Liu, Yue Zhang, Mitchell Yip, Lingzhi Ren, Jialing Liang, Xiupeng Chen, Nan Liu, Ailing Du, Jiaming Wang, Hao Chang, Hyejin Oh, Chen Zhou, Ruxiao Xing, Mengyao Xu, Peiyi Guo, Dominic Gessler, Jun Xie, Phillip W.L. Tai, Guangping Gao, Dan Wang
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 32, Iss 2, Pp 101230- (2024)
Recombinant adeno-associated virus (rAAV)-based gene therapy is entering clinical and commercial stages at an unprecedented pace. Triple transfection of HEK293 cells is currently the most widely used platform for rAAV manufacturing. Here, we develop
Externí odkaz:
https://doaj.org/article/60f1fe4d77694132aa0fbf4e90bcd7e3
Publikováno v:
Zhongguo quanke yixue, Vol 26, Iss 34, Pp 4322-4328 (2023)
Background The emergence of virtual reality (VR) technology is the breakthrough of medical education reform. It is the focus of clinical educators to apply VR technology to medical education and discuss the application effect. The application of VR i
Externí odkaz:
https://doaj.org/article/8706475dd1ad4ec18e47033bffad7b41
Autor:
Manuela Corti, Barry J. Byrne, Dominic J. Gessler, Grace Thompson, Samantha Norman, Jenna Lammers, Kirsten E. Coleman, Cristina Liberati, Melissa E. Elder, Maria L. Escolar, Ibrahim S. Tuna, Clementina Mesaros, Gary I. Kleiner, Deborah S. Barbouth, Heather L. Gray-Edwards, Nathalie Clement, Brian D. Cleaver, Guangping Gao
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 30, Iss , Pp 303-314 (2023)
Gene replacement therapy is a rational therapeutic strategy and clinical intervention for neurodegenerative disorders like Canavan disease, a leukodystrophy caused by biallelic mutations in the aspartoacylase (ASPA) gene. We aimed to investigate whet
Externí odkaz:
https://doaj.org/article/9c3eeeaf5a4542ada6da05e007811039
Autor:
Shashi Bala, Yuan Zhuang, Prashanth Thevkar Nagesh, Donna Catalano, Adam Zivny, Yanbo Wang, Jun Xie, Guangping Gao, Gyongyi Szabo
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 33, Iss , Pp 413-427 (2023)
Most chronic liver diseases progress to liver fibrosis, which, when left untreated, can lead to cirrhosis and hepatocellular carcinoma. MicroRNA (miRNA)-targeted therapeutics have become attractive approaches to treat diseases. In this study, we inve
Externí odkaz:
https://doaj.org/article/d75412b366fa4f0f89a90a39beefcc68
Autor:
Yeon-Suk Yang, Tadatoshi Sato, Sachin Chaugule, Hong Ma, Jun Xie, Guangping Gao, Jae-Hyuck Shim
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 1, Pp 102111- (2024)
Osteogenesis imperfecta (OI) is a genetic disorder characterized by bone fragility, low bone mass, fractures, and extraskeletal manifestations. Since OI is commonly caused by single-nucleotide mutation(s) in the COL1A1 or COL1A2 genes encoding type I
Externí odkaz:
https://doaj.org/article/ec898dcefb954a6485808c098630bb89