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Showing papers on "Genome editing published in 2004"

Genome Editing Using Engineered Nucleases and Their Use in Genomic Screening -- Assay Guidance Manual

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Sittampalam Gs, Grossman A, Brimacombe K, Arkin M, Auld D, Austin C, Baell J, Bejcek B, Caaveiro Jmm, Chung Tdy, Nathan P. Coussens, Dahlin Jl, Devanaryan, Foley Tl, Glicksman M, Hall, Haas Jv, Hoare Srj, Inglese J, Iversen Pw, Kahl Sd, Stephen C. Kales, Kirshner S, Lal-Nag M, Li Z, McGee J, McManus O, Riss T, Saradjian P, Trask Oj, Weidner, Wildey Mj, Xia M, Xu X 
01 Jan 2004

13 citations

Patent•
Methods for production of non-disease causing hemoglobin by ex vivo oligonucleotide gene editing of human stem/progenitor cells

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Wei Han, Matthew J. Chomo, Margaret Wong, Barbara H. Fish, Carolyn M. Ireland, Davette L. Behrens 
30 Apr 2004
TL;DR: The authors applied ex vivo oligonucleotide gene editing to hematopoietic stem cells and/or progenitor cells to create therapeutically effective amounts of wild-type hemoglobin for treatment of the hemoglobinopathies.
Abstract: Methods are presented for applying ex vivo oligonucleotide gene editing to hematopoietic stem cells and/or progenitor cells to create therapeutically effective amounts of wild-type hemoglobin for treatment of the hemoglobinopathies.

5 citations

Journal Article•DOI•
713. Gene Correction Therapy Using Designed Zinc Finger-Based Endonucleases

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Michael C. Holmes, Fyodor D. Urnov, Ya-Li Lee, Jeffrey C. Miller, Philip D. Gregory 
01 May 2004-Molecular Therapy
TL;DR: Data is presented on optimization of the targeting protocol that reproducibly yields > 10' gene-targeted cells in a given population in the absence of any selective pressure, and this approach to gene correction could be used to treat monogenic disorders.

4 citations

Journal Article•DOI•
714. Structure-Based Engineering of the FokI DNA Cleavage Domain in the Context of a Chimeric Endonuclease: Towards Maximally Efficient |[ldquo]|Gene Editing|[rdquo]| Therapy

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Jeffrey C. Miller, Michael C. Holmes, Fyodor D. Urnov, Andrew C. Jamieson, Carl O. Pabo 
01 May 2004-Molecular Therapy
TL;DR: Preliminary experiments using a cell-based gene correction assay indicate that pairs of ZFPs containing modified FokI cleavage domains robustly potentiate the correction of a chromosomally integrated GFP reporter inactivated with a nonsense mutation.

2 citations