Zobrazeno 1 - 10
of 14
pro vyhledávání: '"Jeffrey W. Schindler"'
Autor:
Yildirim Dogan, Cecilia N. Barese, Jeffrey W. Schindler, John K. Yoon, Zeenath Unnisa, Swaroopa Guda, Mary E. Jacobs, Christine Oborski, Tim Maiwald, Diana L. Clarke, Axel Schambach, Richard Pfeifer, Claudia Harper, Chris Mason, Niek P. van Til
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 27, Iss , Pp 464-487 (2022)
Pompe disease is a rare genetic neuromuscular disorder caused by acid α-glucosidase (GAA) deficiency resulting in lysosomal glycogen accumulation and progressive myopathy. Enzyme replacement therapy, the current standard of care, penetrates poorly i
Externí odkaz:
https://doaj.org/article/19aee40652b94df1a4979d0e41238f5c
Publikováno v:
Biomedicines, Vol 10, Iss 2, p 302 (2022)
Pompe disease is an inherited neuromuscular disorder caused by deficiency of the lysosomal enzyme acid alpha-glucosidase (GAA). The most severe form is infantile-onset Pompe disease, presenting shortly after birth with symptoms of cardiomyopathy, res
Externí odkaz:
https://doaj.org/article/c76acd8f3abf4980b14f9ce876629d2b
Autor:
Robert N. Plasschaert, Mark P. DeAndrade, Fritz Hull, Quoc Nguyen, Tara Peterson, Aimin Yan, Mariana Loperfido, Cristina Baricordi, Luigi Barbarossa, John K. Yoon, Yildirim Dogan, Zeenath Unnisa, Jeffrey W. Schindler, Niek P. van Til, Luca Biasco, Chris Mason
Publikováno v:
Molecular Therapy. 30:3209-3225
Hematopoietic stem/progenitor cell gene therapy (HSPC-GT) has shown clear neurological benefit in rare diseases, which is achieved through the engraftment of genetically modified microglia-like cells (MLCs) in the brain. Still, the engraftment dynami
Autor:
Yildirim Dogan, Cecilia N. Barese, Jeffrey W. Schindler, John K. Yoon, Zeenath Unnisa, Swaroopa Guda, Mary E. Jacobs, Christine Oborski, Diana L. Clarke, Axel Schambach, Richard Pfeifer, Claudia Harper, Chris Mason, Niek P. van Til
Pompe disease is a rare genetic neuromuscular disorder caused by acid alpha-glucosidase (GAA) deficiency resulting in lysosomal glycogen accumulation and progressive myopathy. Enzyme replacement therapy (ERT) is the current standard of care, which pr
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::89f466bcec75e33a143d065b6b9e12b3
https://doi.org/10.1101/2021.12.28.474352
https://doi.org/10.1101/2021.12.28.474352
Genetically engineered microglia-like cells have therapeutic potential for neurodegenerative disease
Autor:
Luigi Barbarossa, Niek P. van Til, Tara Peterson, Cristina Baricordi, Christopher E. Mason, Robert N. Plasschaert, Zeenath Unnisa, Mariana Loperfido, Jeffrey W. Schindler, Aimin Yan, Fritz Hull, Yildirim Dogan, Mark P. DeAndrade, John K. Yoon, Luca Biasco, Quoc Nguyen
Hematopoietic stem/progenitor cell gene therapy (HSPC-GT) results in the engraftment of genetically modified microglia-like cells (MLCs) in the brain. While HSPC-GT has shown a clear neurological benefit in the clinic for specific rare diseases, the
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::1f54bc3f10bedc2465a41f5e0e4cb191
https://doi.org/10.1101/2021.09.16.460703
https://doi.org/10.1101/2021.09.16.460703
Autor:
Niek P. van Til, Jeffrey W. Schindler, John K. Yoon, Yildirim Dogan, Cecilia Barese, Zeenath Unnisa, Swaroopa Guda, Mary E. Jacobs, Daniella Pizzurro, Mirjam Trame, Tim Maiwald, Christine Oborski, Michael Rothe, Axel Schambach, Claudia Harper, Richard Pfeifer, Chris Mason
Publikováno v:
Molecular Genetics and Metabolism. 135:S122-S123
Autor:
Hui Liu, Forest M. White, Richard O. Hynes, Shao En Ong, Kwabena Badu-Nkansah, Jeffrey W. Schindler
Publikováno v:
Proceedings of the National Academy of Sciences. 108:1379-1384
We report the application of quantitative mass spectrometry to identify plasma membrane proteins differentially expressed in melanoma cells with high vs. low metastatic abilities. Using stable isotope labeling with amino acids in culture (SILAC) coup
Autor:
Jeffrey W. Schindler, Hanno Hock, Adlen Foudi, Denille Van Buren, Konrad Hochedlinger, Vincent J. Carey, Rudolf Jaenisch
Publikováno v:
Nature biotechnology
Hematopoietic stem cells (HSCs) are thought to divide infrequently based on their resistance to cytotoxic injury targeted at rapidly cycling cells1, 2 and have been presumed to retain labels such as the nucleotide analogue 5-bromodeoxyuridine (BrdU).
Autor:
Jeffrey W. Schindler, Hanno Hock, Heather M. Rooke, Yuko Fujiwara, Stuart H. Orkin, Shireen Saleque, Melanie J. Hamblen
Publikováno v:
Nature. 431:1002-1007
Haematopoietic stem cells (HSCs) sustain blood production throughout life. HSCs are capable of extensive proliferative expansion, as a single HSC may reconstitute lethally irradiated hosts1. In steady-state, HSCs remain largely quiescent and self-ren
Publikováno v:
Proceedings of the National Academy of Sciences of the United States of America. 109(37)
The transcriptional coactivator Yes-associated protein (YAP) is a major regulator of organ size and proliferation in vertebrates. As such, YAP can act as an oncogene in several tissue types if its activity is increased aberrantly. Although no activat