Zobrazeno 1 - 10
of 386
pro vyhledávání: '"Adeno associated viral vectors"'
Autor:
Alberto J. Rico, Almudena Corcho, Julia Chocarro, Goiaz Ariznabarreta, Elvira Roda, Adriana Honrubia, Patricia Arnaiz, José L. Lanciego
Publikováno v:
Frontiers in Neuroanatomy, Vol 18 (2024)
IntroductionThe presence of a widespread cortical synucleinopathy is the main neuropathological hallmark underlying clinical entities such as Parkinson’s disease with dementia (PDD) and dementia with Lewy bodies (DLB). There currently is a pressing
Externí odkaz:
https://doaj.org/article/6a96e6f1b3464063b996feb48b48365a
Autor:
Bijina Balakrishnan, Xinhua Yan, Marshall D. McCue, Olivia Bellagamba, Aaron Guo, Felicity Winkler, Jason Thall, Lisa Crawford, Rain Dimen, Sara Chen, Sean McEnaney, Yiman Wu, Mike Zimmer, Joe Sarkis, Paolo G.V. Martini, Patrick F. Finn, Kent Lai
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 32, Iss 1, Pp 101191- (2024)
Despite the implementation of lifesaving newborn screening programs and a galactose-restricted diet, many patients with classic galactosemia develop long-term debilitating neurological deficits and primary ovarian insufficiency. Previously, we showed
Externí odkaz:
https://doaj.org/article/d2fda7725fe9434a86e2d785a3e6d368
Publikováno v:
Current Issues in Molecular Biology, Vol 45, Iss 10, Pp 7705-7720 (2023)
CAR-T cell therapy involves genetically engineering T cells to recognize and attack tumour cells by adding a chimeric antigen receptor (CAR) to their surface. In this study, we have used dual transduction with AAV serotype 6 (AAV6) to integrate an an
Externí odkaz:
https://doaj.org/article/f4da53d5c7cf42108b22e8ca2b4715fd
AAV-CRISPR-Cas13 eliminates human enterovirus and prevents death of infected miceResearch in context
Autor:
Choong Tat Keng, Thinesshwary Yogarajah, Regina Ching Hua Lee, Irfan Bin Hajis Muhammad, Bing Shao Chia, Suraj Rajan Vasandani, Daryl Shern Lim, Ke Guo, Yi Hao Wong, Chee Keng Mok, Justin Jang Hann Chu, Wei Leong Chew
Publikováno v:
EBioMedicine, Vol 93, Iss , Pp 104682- (2023)
Summary: Background: RNA viruses account for many human diseases and pandemic events but are often not targetable by traditional therapeutics modalities. Here, we demonstrate that adeno-associated virus (AAV) -delivered CRISPR-Cas13 directly targets
Externí odkaz:
https://doaj.org/article/d913a4522d6244fb8aee77bc8780cbd7
Autor:
Ridhwaanah Jacobs, Makafui Dennis Dogbey, Njabulo Mnyandu, Keila Neves, Stefan Barth, Patrick Arbuthnot, Mohube Betty Maepa
Publikováno v:
Microorganisms, Vol 11, Iss 12, p 2985 (2023)
Hepatitis B virus (HBV) has afflicted humankind for decades and there is still no treatment that can clear the infection. The development of recombinant adeno-associated virus (rAAV)-based gene therapy for HBV infection has become important in recent
Externí odkaz:
https://doaj.org/article/d0c6413a36ca492399e5c5715e39fed7
Akademický článek
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Autor:
Chiara Peres, Caterina Sellitto, Chiara Nardin, Sabrina Putti, Tiziana Orsini, Chiara Di Pietro, Daniela Marazziti, Adriana Vitiello, Arianna Calistri, Mara Rigamonti, Ferdinando Scavizzi, Marcello Raspa, Francesco Zonta, Guang Yang, Thomas W. White, Fabio Mammano
Publikováno v:
EBioMedicine, Vol 89, Iss , Pp 104453- (2023)
Summary: Background: Keratitis ichthyosis deafness (KID) syndrome is a rare disorder caused by hemichannel (HC) activating gain-of-function mutations in the GJB2 gene encoding connexin (Cx) 26, for which there is no cure, or current treatments based
Externí odkaz:
https://doaj.org/article/bd169d1992394aeab17af27a91b30026
Autor:
Fatima Aerts-Kaya, Niek P. van Til
Publikováno v:
Pharmaceutics, Vol 15, Iss 11, p 2522 (2023)
Leukodystrophies are a heterogenous group of inherited, degenerative encephalopathies, that if left untreated, are often lethal at an early age. Although some of the leukodystrophies can be treated with allogeneic hematopoietic stem cell transplantat
Externí odkaz:
https://doaj.org/article/ca0f9b3814034bc8a610a1e73c752ec3
Autor:
Lillian J. Campos, Cynthia M. Arokiaraj, Miguel R. Chuapoco, Xinhong Chen, Nick Goeden, Viviana Gradinaru, Andrew S. Fox
Publikováno v:
Current Research in Neurobiology, Vol 4, Iss , Pp 100086- (2023)
Modern neuroscience approaches including optogenetics, calcium imaging, and other genetic manipulations have facilitated our ability to dissect specific circuits in rodent models to study their role in neurological disease. These approaches regularly
Externí odkaz:
https://doaj.org/article/a1d130913dd94fb9958f2b34f0a4719d
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