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Akademický článek
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Autor:
Christopher R. Barnes, Hyuncheol Lee, David S. Ojala, Kazuomori K. Lewis, Prajit Limsirichai, David V. Schaffer
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 26, Iss , Pp 94-103 (2021)
We describe a genome-wide screening strategy to identify target genes whose modulation increases the capacity of a cell to produce recombinant adeno-associated viral (AAV) vector. Specifically, a single-guide RNA (sgRNA) library for a CRISPR-based ge
Externí odkaz:
https://doaj.org/article/187555a0fb554eeea40ee4cf01283699
Publikováno v:
iScience, Vol 25, Iss 10, Pp 104971- (2022)
Summary: Human pluripotent stem cells (hPSCs) are an exciting and promising source to enable cell replacement therapies for a variety of unmet medical needs. Though hPSCs can be successfully derived into numerous physiologically relevant cell types,
Externí odkaz:
https://doaj.org/article/5a9e58c310cb4bfa8f45c124771cc0e1
Akademický článek
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K zobrazení výsledku je třeba se přihlásit.
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Publikováno v:
Frontiers in Cellular Neuroscience, Vol 16 (2022)
Cells reside in a dynamic microenvironment that presents them with regulatory signals that vary in time, space, and amplitude. The cell, in turn, interprets these signals and accordingly initiates downstream processes including cell proliferation, di
Externí odkaz:
https://doaj.org/article/f0365195bb614510aba731b48c966b8a
Autor:
Bilge E Öztürk, Molly E Johnson, Michael Kleyman, Serhan Turunç, Jing He, Sara Jabalameli, Zhouhuan Xi, Meike Visel, Valérie L Dufour, Simone Iwabe, Luis Felipe L Pompeo Marinho, Gustavo D Aguirre, José-Alain Sahel, David V Schaffer, Andreas R Pfenning, John G Flannery, William A Beltran, William R Stauffer, Leah C Byrne
Publikováno v:
eLife, Vol 10 (2021)
Background: Adeno-associated virus (AAV)-mediated gene therapies are rapidly advancing to the clinic, and AAV engineering has resulted in vectors with increased ability to deliver therapeutic genes. Although the choice of vector is critical, quantita
Externí odkaz:
https://doaj.org/article/2f839da7c7b643a2acc12975971ae2e2
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 18, Iss , Pp 432-443 (2019)
Interfacing gene delivery vehicles with biomaterials has the potential to play a key role in diversifying gene transfer capabilities, including localized, patterned, and controlled delivery. However, strategies for modifying biomaterials to interact
Externí odkaz:
https://doaj.org/article/c81580fbc60145aaa290c025a95b2d5b
Autor:
Freja K. Ekman, David S. Ojala, Maroof M. Adil, Paola A. Lopez, David V. Schaffer, Thomas Gaj
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 17, Iss , Pp 829-839 (2019)
Huntington’s disease (HD) is a currently incurable and, ultimately, fatal neurodegenerative disorder caused by a CAG trinucleotide repeat expansion within exon 1 of the huntingtin (HTT) gene, which results in the production of a mutant protein that
Externí odkaz:
https://doaj.org/article/9124d693316b4ce19eb68f74da73bd30
Publikováno v:
STAR Protocols, Vol 1, Iss 3, Pp 100141- (2020)
Summary: Optogenetic modulation of protein interactions enables spatiotemporal control of cellular signaling dynamics in a variety of biological systems. However, light patterning by standard microscopes is limited by their complexity, sample through
Externí odkaz:
https://doaj.org/article/fa4fd3b39b1b4c48a14e8d5544428f44
Publikováno v:
iScience, Vol 6, Iss , Pp 13-21 (2018)
Summary: Despite extensive research on the canonical Wnt signaling pathway, the mechanism by which this signal downregulates the activity of destruction complexes and inhibits β-catenin degradation remains controversial. In particular, recent attent
Externí odkaz:
https://doaj.org/article/086e4e33303a405ba9ae82a876a76eba