Zobrazeno 1 - 5
of 5
pro vyhledávání: '"Charlotte Platner"'
Publikováno v:
PLoS ONE, Vol 9, Iss 8, p e104022 (2014)
Efficient ex vivo transduction of hematopoietic stem cells (HSCs) is encumbered by differentiation which reduces engraftment. We hypothesized that inhibiting DNA methyltransferase with decitabine would block differentiation of transduced CD34+ cells
Externí odkaz:
https://doaj.org/article/72e12464501a4973ace3fece1286ea51
Autor:
John F. Tisdale, Brian Mozer, Kareem Washington, Luke P Skala, Lloyd G Mitchell, Lydia N. Raines, Naoya Uchida, Anna Shvygin, Matthew M. Hsieh, Charlotte Platner, Josiah Ballantine
Publikováno v:
Human gene therapy methods. 28(2)
Sickle cell disease results from a point mutation in exon 1 of the β-globin gene (total 3 exons). Replacing sickle β-globin exon 1 (and exon 2) with a normal sequence by trans-splicing is a potential therapeutic strategy. Therefore, this study soug
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::1942a222fdfb942a292b7b8de57e1ca1
https://pubmed.ncbi.nlm.nih.gov/28267358
https://pubmed.ncbi.nlm.nih.gov/28267358
Autor:
Matthew M. Hsieh, Lloyd G. Mitchell, Naoya Uchida, Brian Mozer, Kareem Washington, Charlotte Platner, Josiah Ballantine, John F. Tisdale
Publikováno v:
Molecular Therapy. 23
Sickle cell disease is caused by a point mutation in exon 1 of the β-globin gene (3 exons and 2 introns). RNA splicing between two distinct pieces of pre-mRNA, known as trans-splicing, represents a potential therapeutic strategy allowing replacement
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::752ed5693f747aa81c21070f9b9e7e95
Publikováno v:
PLoS ONE
PLoS ONE, Vol 9, Iss 8, p e104022 (2014)
PLoS ONE, Vol 9, Iss 8, p e104022 (2014)
Efficient ex vivo transduction of hematopoietic stem cells (HSCs) is encumbered by differentiation which reduces engraftment. We hypothesized that inhibiting DNA methyltransferase with decitabine would block differentiation of transduced CD34+ cells
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::864d64c258a5fdff6af01a08126abe9d
https://pubmed.ncbi.nlm.nih.gov/25089909
https://pubmed.ncbi.nlm.nih.gov/25089909
Autor:
Josiah Ballantine, Naoya Uchida, R. Patrick Weitzel, Robert E. Donahue, John F. Tisdale, Aylin C. Bonifacino, Sandra D. Price, Charlotte Platner, Allen E. Krouse, Mark E. Metzger
Publikováno v:
Molecular Therapy. 23:S118-S119
Reduced intensity conditioning (RIC) regimens are desirable for hematopoietic stem cell (HSC)-targeted gene therapy. However, in previous gene therapy trials, low gene marking was reported in peripheral blood cells, raising the question that RIC migh
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::e87d2c57f93ef6bb94825be8b514478e