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Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 32, Iss 1, Pp 101211- (2024)
Intra-articular adeno-associated virus (AAV) gene therapy has been explored as a potential strategy for joint diseases. However, concerns of low transduction efficacy, off-target expression, and neutralizing antibodies (Nabs) still need to be address
Externí odkaz:
https://doaj.org/article/6b63d18ea2c14b95ade6ad56c358ead2
Autor:
Caroline Le Guiner, Xiao Xiao, Thibaut Larcher, Aude Lafoux, Corinne Huchet, Gilles Toumaniantz, Oumeya Adjali, Ignacio Anegon, Séverine Remy, Josh Grieger, Juan Li, Vahid Farrokhi, Hendrik Neubert, Jane Owens, Maritza McIntyre, Philippe Moullier, R. Jude Samulski
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 30, Iss , Pp 30-47 (2023)
Duchenne muscular dystrophy (DMD) is an X-linked disease caused by loss-of-function mutations in the dystrophin gene and is characterized by muscle wasting and early mortality. Adeno-associated virus-mediated gene therapy is being investigated as a t
Externí odkaz:
https://doaj.org/article/7a89e1515ecf4452944c4318d3851f25
Autor:
Trang Nguyen Kieu Doan, Matthew D. Le, Irnela Bajrovic, Lorne Celentano, Charles Krause, Haley Grooms Balyan, Abbie Svancarek, Angela Mote, Anna Tretiakova, R. Jude Samulski, Maria A. Croyle
Publikováno v:
Communications Medicine, Vol 2, Iss 1, Pp 1-14 (2022)
Doan et al. characterize and assess the stability of film matrix embedded Adeno-Associated Virus 9 (AAV9) vectors during storage and transport at ambient temperatures. High and low viscosity formulations of AAV9 stored at 25 °C maintain titer for 6
Externí odkaz:
https://doaj.org/article/77c089e3d0584aa399759a4651c15e4a
Autor:
Parvathy Thampi, R. Jude Samulski, Joshua C. Grieger, Jennifer N. Phillips, C. Wayne McIlwraith, Laurie R. Goodrich
Publikováno v:
Frontiers in Veterinary Science, Vol 9 (2023)
Externí odkaz:
https://doaj.org/article/833bace64d6e4417b92cf39058342837
Autor:
Parvathy Thampi, R. Jude Samulski, Joshua C. Grieger, Jennifer N. Phillips, C. Wayne McIlwraith, Laurie R. Goodrich
Publikováno v:
Frontiers in Veterinary Science, Vol 9 (2022)
With an intrinsically low ability for self-repair, articular cartilage injuries often progress to cartilage loss and joint degeneration resulting in osteoarthritis (OA). Osteoarthritis and the associated articular cartilage changes can be debilitatin
Externí odkaz:
https://doaj.org/article/d56916efcfad4d71a6453d01888fc341
Autor:
Jeremy S. Francis, Vladimir Markov, Irenuez D. Wojtas, Steve Gray, Thomas McCown, R. Jude Samulski, Marciano Figueroa, Paola Leone
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 20, Iss , Pp 520-534 (2021)
Recent advances in adeno-associated viral (AAV) capsid variants with novel oligotropism require validation in models of disease in order to be viable candidates for white matter disease gene therapy. We present here an assessment of the biodistributi
Externí odkaz:
https://doaj.org/article/b01784b4f1824fefaae134f8465d61b6
Autor:
Xiaolei Pei, Wenwei Shao, Allene Xing, Charles Askew, Xiaojing Chen, Caibin Cui, Yasmina L. Abajas, David A. Gerber, Elizabeth P. Merricks, Timothy C. Nichols, Wuping Li, R. Jude Samulski, Chengwen Li
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 18, Iss , Pp 259-268 (2020)
Adeno-associated virus (AAV) vectors have been successfully used in patients with bleeding disorders and blindness. For human liver targeting, two major factors restrict effective AAV transduction after systemic administration of AAV vectors: human h
Externí odkaz:
https://doaj.org/article/ffd1eccee8e6456aa953cf4176aa8c2d
Autor:
Wenwei Shao, Junjiang Sun, Xiaojing Chen, Amanda Dobbins, Elizabeth P. Merricks, R. Jude Samulski, Timothy C. Nichols, Chengwen Li
Publikováno v:
Frontiers in Pharmacology, Vol 13 (2022)
Adeno-associated virus (AAV) mediated gene therapy has been successfully applied in clinical trials, including hemophilia. Novel AAV vectors have been developed with enhanced transduction and specific tissue tropism. Considering the difference in eff
Externí odkaz:
https://doaj.org/article/2afe3a173d2f462ca9c1fcf4aa273b71
Autor:
Tierney B. Daw, Hala G. El-Nahal, Michele A. Basso, Elizabeth J. Jun, Alex R. Bautista, R. Jude Samulski, Marc A. Sommer, Martin O. Bohlen
Publikováno v:
Human Gene Therapy. 34:228-246
Viral vector technologies are commonly used in neuroscience research to understand and manipulate neural circuits, but successful applications of these technologies in non-human primate models have been inconsistent. An essential component to improve
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::9290890a59850714f504a63560f3befd
https://doi.org/10.1089/hum.2022.194
https://doi.org/10.1089/hum.2022.194