Výsledky vyhledávání - "Edward J, Rebar"

Akademický článek

Diversifying the structure of zinc finger nucleases for high-precision genome editing

Autor: David E. Paschon, Stephanie Lussier, Tenzin Wangzor, Danny F. Xia, Patrick W. Li, Sarah J. Hinkley, Nicholas A. Scarlott, Stephen C. Lam, Adam J. Waite, Lynn N. Truong, Nimisha Gandhi, Bhakti N. Kadam, Deepak P. Patil, David A. Shivak, Gary K. Lee, Michael C. Holmes, Lei Zhang, Jeffrey C. Miller, Edward J. Rebar

Publikováno v: Nature Communications, Vol 10, Iss 1, Pp 1-12 (2019)

Genome editing often requires cleavage within a narrow sequence window. Here the authors develop an expanded set of zinc finger nuclease architectures that increase the available configurations by a factor of 64 and can target almost every base at lo

Externí odkaz: https://doaj.org/article/52399a6682884a7196ea64923e436811

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Akademický článek

Long-Term Engraftment and Fetal Globin Induction upon BCL11A Gene Editing in Bone-Marrow-Derived CD34+ Hematopoietic Stem and Progenitor Cells

Publikováno v: Molecular Therapy: Methods & Clinical Development, Vol 4, Iss C, Pp 137-148 (2017)

To develop an effective and sustainable cell therapy for sickle cell disease (SCD), we investigated the feasibility of targeted disruption of the BCL11A gene, either within exon 2 or at the GATAA motif in the intronic erythroid-specific enhancer, usi

Externí odkaz: https://doaj.org/article/78e274bd1cdc4349b7f3df729d3543f4

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Akademický článek

Gene Correction of iPSCs from a Wiskott-Aldrich Syndrome Patient Normalizes the Lymphoid Developmental and Functional Defects

Autor: Tamara J. Laskowski, Yasmine Van Caeneghem, Rasoul Pourebrahim, Chao Ma, Zhenya Ni, Zita Garate, Ana M. Crane, Xuan Shirley Li, Wei Liao, Manuel Gonzalez-Garay, Jose Carlos Segovia, David E. Paschon, Edward J. Rebar, Michael C. Holmes, Dan Kaufman, Bart Vandekerckhove, Brian R. Davis

Publikováno v: Stem Cell Reports, Vol 7, Iss 2, Pp 139-148 (2016)

Wiskott-Aldrich syndrome (WAS) is an X-linked primary immunodeficiency disease caused by mutations in the gene encoding the WAS protein (WASp). Here, induced pluripotent stem cells (iPSCs) were derived from a WAS patient (WAS-iPSC) and the endogenous

Externí odkaz: https://doaj.org/article/676abc2202b146eb97e086edfc92f229

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Akademický článek

Multi-reporter selection for the design of active and more specific zinc-finger nucleases for genome editing

Autor: Benjamin L. Oakes, Danny F. Xia, Elizabeth F. Rowland, Denise J. Xu, Irina Ankoudinova, Jennifer S. Borchardt, Lei Zhang, Patrick Li, Jeffrey C. Miller, Edward J. Rebar, Marcus B. Noyes

Publikováno v: Nature Communications, Vol 7, Iss 1, Pp 1-13 (2016)

Zinc finger nucleases have an established role in genome editing. Here, the authors report a strategy for identifying zinc finger nucleases that discriminate between desired targets and provide genome-wide specificity.

Externí odkaz: https://doaj.org/article/e66bba0eeef149b0809ce032052bdf58

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Akademický článek

Human Intestinal Tissue with Adult Stem Cell Properties Derived from Pluripotent Stem Cells

Autor: Ryan Forster, Kunitoshi Chiba, Lorian Schaeffer, Samuel G. Regalado, Christine S. Lai, Qing Gao, Samira Kiani, Henner F. Farin, Hans Clevers, Gregory J. Cost, Andy Chan, Edward J. Rebar, Fyodor D. Urnov, Philip D. Gregory, Lior Pachter, Rudolf Jaenisch, Dirk Hockemeyer

Publikováno v: Stem Cell Reports, Vol 2, Iss 6, Pp 838-852 (2014)

Genetically engineered human pluripotent stem cells (hPSCs) have been proposed as a source for transplantation therapies and are rapidly becoming valuable tools for human disease modeling. However, many applications are limited due to the lack of rob

Externí odkaz: https://doaj.org/article/29251974b44c49fb94543dca7d398aa3

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Akademický článek

Mitochondrially targeted ZFNs for selective degradation of pathogenic mitochondrial genomes bearing large‐scale deletions or point mutations

Autor: Payam A Gammage, Joanna Rorbach, Anna I Vincent, Edward J Rebar, Michal Minczuk

Publikováno v: EMBO Molecular Medicine, Vol 6, Iss 4, Pp 458-466 (2014)

Abstract We designed and engineered mitochondrially targeted obligate heterodimeric zinc finger nucleases (mtZFNs) for site‐specific elimination of pathogenic human mitochondrial DNA (mtDNA). We used mtZFNs to target and cleave mtDNA harbouring the

Externí odkaz: https://doaj.org/article/1f20643c6a5a43d8b1fa075b77589992

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Enhancing gene editing specificity by attenuating DNA cleavage kinetics

Autor: Andreas Reik, Sarah J. Hinkley, Nicholas A. Scarlott, Deepak P. Patil, Yuri R. Bendana, David A. Shivak, Tenzin Wangzor, Stephen Lam, Yuanyue Zhou, David Paschon, Lei Zhang, Patrick Li, Friedrich Fauser, Gary Lee, Jeffrey C. Miller, Charles B Paine, Danny F Xia, Edward J. Rebar, Hunter W. Richards

Publikováno v: Nature Biotechnology. 37:945-952

Engineered nucleases have gained broad appeal for their ability to mediate highly efficient genome editing. However the specificity of these reagents remains a concern, especially for therapeutic applications, given the potential mutagenic consequenc

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c8cc9ae3ffe9baf7fec7a19c03368407
https://doi.org/10.1038/s41587-019-0186-z

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Abstract 5598: Engineered hypoimmune allogeneic CAR T cells as potential off-the-shelf CAR T cell immunotherapies

Publikováno v: Cancer Research. 82:5598-5598

Off-the-shelf CAR T cells may offer advantages over autologous strategies, including ease of manufacturing, improved quality control with avoidance of malignant contamination and T cell dysfunction, and the ability to generate a final product from he

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::3da74a1da1f85fe790561b43db5502a0
https://doi.org/10.1158/1538-7445.am2022-5598

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