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https://www.cell.com/molecular-therapy-family/molecular-therapy/fulltext/S1525-0016(18)30019-4
Apr 04, 2018 · The Sleeping Beauty (SB) transposon system is a non-viral gene delivery platform that combines simplicity, inexpensive manufacture, and favorable safety features in the context of human applications. However, efficient correction of hematopoietic stem and progenitor cells (HSPCs) with non-viral vector systems, including SB,...Author: Marta Holstein, Cristina Mesa-Nuñez, Csaba Miskey, Elena Almarza, Valentina Poletti, Marco Schmeer, ...
https://www.ncbi.nlm.nih.gov/pubmed/29503198
Apr 04, 2018 · Efficient Non-viral Gene Delivery into Human Hematopoietic Stem Cells by Minicircle Sleeping Beauty Transposon Vectors.Author: Marta Holstein, Cristina Mesa-Nuñez, Csaba Miskey, Elena Almarza, Valentina Poletti, Marco Schmeer, ...
https://www.nature.com/articles/1704081
Jun 23, 2003 · Most current viral gene therapy vectors targeting hematopoietic stem cells are modified in a number of key ways to make them safe for clinical use. First, the vectors are ‘gutted’ of the endogenous viral genes necessary for viral replication and budding from the cell.Author: J L Vollweiler, S P Zielske, J S Reese, S L Gerson
https://www.cell.com/molecular-therapy-family/molecular-therapy/pdfExtended/S1525-0016(18)30019-4
gene delivery platform that combines simplicity, inexpensive manufacture, and favorable safety features in the context of human applications. However, efficient correction of hematopoietic stem and progenitor cells (HSPCs) with non-viral vector systems, including SB, demands further refine-ment of gene delivery techniques. We set out to improve
https://www.nature.com/articles/s41573-020-0064-x
Insertion of a normal, wild-type WAS gene into a patient’s haematopoietic stem and progenitor cells may thus be able to functionally compensate for their mutated gene upon their differentiation ...
https://www.ncbi.nlm.nih.gov/pubmed/31416800
Aug 15, 2019 · Therapeutic gene delivery to hematopoietic stem cells (HSCs) holds great potential as a life-saving treatment for a range of monogenic, oncologic, and infectious diseases. However, clinical gene therapy is severely limited by intrinsic HSC resistance to modification with lentiviral vectors (LVs), thus requiring high doses or repeat LV administration to achieve therapeutic gene correction.Author: Stosh Ozog, Nina D. Timberlake, Kip Hermann, Olivia Garijo, Kevin G. Haworth, Guoli Shi, Christopher...
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