Multiplex HDR for disease and correction modeling of SCID by CRISPR genome editing in human HSPCs

Autor: Ortal Iancu, Daniel Allen, Orli Knop, Yonathan Zehavi, Dor Breier, Adaya Arbiv, Atar Lev, Yu Nee Lee, Katia Beider, Arnon Nagler, Raz Somech, Ayal Hendel

Publikováno v: Molecular Therapy: Nucleic Acids, Vol 31, Iss , Pp 105-121 (2023)

Severe combined immunodeficiency (SCID) is a group of disorders caused by mutations in genes involved in the process of lymphocyte maturation and function. CRISPR-Cas9 gene editing of the patient’s own hematopoietic stem and progenitor cells (HSPCs

Externí odkaz: https://doaj.org/article/ae155571bbed4c078012ef702c2c303a

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Integrated Glycoproteomics Identifies a Role of N-Glycosylation and Galectin-1 on Myogenesis and Muscle Development

Autor: Morten Thaysen-Andersen, Deanne Francis, Gregory A. Quaife-Ryan, Long H. Chung, Pengyi Yang, Kevin I. Watt, Jodie L. Abrahams, Christopher Ashwood, Hongwei Qian, Benjamin L. Parker, Paul Gregorevic, Ronnie Blazev, James E. Hudson

Publikováno v: Molecular & Cellular Proteomics : MCP

Many cell surface and secreted proteins are modified by the covalent addition of glycans that play an important role in the development of multicellular organisms. These glycan modifications enable communication between cells and the extracellular ma

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f877e8a75bfb74afac377475bd0c1016
https://doi.org/10.1074/mcp.ra120.002166

Multiplex HDR for disease and correction modeling of SCID by CRISPR genome editing in human HSPCs.

Autor: Iancu O; The Institute for Advanced Materials and Nanotechnology, The Mina and Everard Goodman Faculty of Life Sciences, Bar-Ilan University, Ramat Gan 5290002, Israel., Allen D; The Institute for Advanced Materials and Nanotechnology, The Mina and Everard Goodman Faculty of Life Sciences, Bar-Ilan University, Ramat Gan 5290002, Israel., Knop O; The Institute for Advanced Materials and Nanotechnology, The Mina and Everard Goodman Faculty of Life Sciences, Bar-Ilan University, Ramat Gan 5290002, Israel., Zehavi Y; The Institute for Advanced Materials and Nanotechnology, The Mina and Everard Goodman Faculty of Life Sciences, Bar-Ilan University, Ramat Gan 5290002, Israel., Breier D; The Institute for Advanced Materials and Nanotechnology, The Mina and Everard Goodman Faculty of Life Sciences, Bar-Ilan University, Ramat Gan 5290002, Israel., Arbiv A; The Institute for Advanced Materials and Nanotechnology, The Mina and Everard Goodman Faculty of Life Sciences, Bar-Ilan University, Ramat Gan 5290002, Israel., Lev A; The Institute for Advanced Materials and Nanotechnology, The Mina and Everard Goodman Faculty of Life Sciences, Bar-Ilan University, Ramat Gan 5290002, Israel.; Pediatric Department A and the Immunology Service, Jeffrey Modell Foundation Center, Edmond and Lily Safra Children's Hospital, Sheba Medical Center, Tel-HaShomer, Ramat Gan 5266202, Israel., Lee YN; Pediatric Department A and the Immunology Service, Jeffrey Modell Foundation Center, Edmond and Lily Safra Children's Hospital, Sheba Medical Center, Tel-HaShomer, Ramat Gan 5266202, Israel.; Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv 6997801, Israel., Beider K; The Division of Hematology and Bone Marrow Transplantation, Chaim Sheba Medical Center, Tel-HaShomer, Ramat Gan 5266202, Israel., Nagler A; Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv 6997801, Israel.; The Division of Hematology and Bone Marrow Transplantation, Chaim Sheba Medical Center, Tel-HaShomer, Ramat Gan 5266202, Israel., Somech R; Pediatric Department A and the Immunology Service, Jeffrey Modell Foundation Center, Edmond and Lily Safra Children's Hospital, Sheba Medical Center, Tel-HaShomer, Ramat Gan 5266202, Israel.; Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv 6997801, Israel., Hendel A; The Institute for Advanced Materials and Nanotechnology, The Mina and Everard Goodman Faculty of Life Sciences, Bar-Ilan University, Ramat Gan 5290002, Israel.

Publikováno v: Molecular therapy. Nucleic acids [Mol Ther Nucleic Acids] 2022 Dec 09; Vol. 31, pp. 105-121. Date of Electronic Publication: 2022 Dec 09 (Print Publication: 2023).