Targeted knock-in of NCF1 cDNA into the NCF2 locus leads to myeloid phenotypic correction of p47 phox -deficient chronic granulomatous disease.

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Title: Targeted knock-in of NCF1 cDNA into the NCF2 locus leads to myeloid phenotypic correction of p47 phox -deficient chronic granulomatous disease.
Authors: Siow KM; Division of Gene and Cell Therapy, Institute for Regenerative Medicine, University of Zurich, Schlieren, 8952 Zurich, Switzerland., Güngör M; Division of Gene and Cell Therapy, Institute for Regenerative Medicine, University of Zurich, Schlieren, 8952 Zurich, Switzerland., Wrona D; Division of Gene and Cell Therapy, Institute for Regenerative Medicine, University of Zurich, Schlieren, 8952 Zurich, Switzerland., Raimondi F; Division of Gene and Cell Therapy, Institute for Regenerative Medicine, University of Zurich, Schlieren, 8952 Zurich, Switzerland., Pastukhov O; Division of Gene and Cell Therapy, Institute for Regenerative Medicine, University of Zurich, Schlieren, 8952 Zurich, Switzerland., Tsapogas P; Division of Gene and Cell Therapy, Institute for Regenerative Medicine, University of Zurich, Schlieren, 8952 Zurich, Switzerland., Menzi T; Division of Gene and Cell Therapy, Institute for Regenerative Medicine, University of Zurich, Schlieren, 8952 Zurich, Switzerland., Schmitz M; Department of Biochemistry, University of Zurich, 8057 Zurich, Switzerland., Kulcsár PI; Institute of Pharmacology and Toxicology, University of Zurich, 8057 Zurich, Switzerland., Schwank G; Institute of Pharmacology and Toxicology, University of Zurich, 8057 Zurich, Switzerland., Schulz A; Department of Pediatrics, University Medical Center Ulm, 89075 Ulm, Germany., Jinek M; Department of Biochemistry, University of Zurich, 8057 Zurich, Switzerland., Modlich U; Division of Gene and Cell Therapy, Institute for Regenerative Medicine, University of Zurich, Schlieren, 8952 Zurich, Switzerland., Siler U; Division of Gene and Cell Therapy, Institute for Regenerative Medicine, University of Zurich, Schlieren, 8952 Zurich, Switzerland.; School of Life Sciences, Institute for Pharma Technology, University of Applied Sciences and Arts Northwestern Switzerland, 4132 Muttenz, Switzerland., Reichenbach J; Division of Gene and Cell Therapy, Institute for Regenerative Medicine, University of Zurich, Schlieren, 8952 Zurich, Switzerland.; Department of Somatic Gene Therapy, University Children's Hospital Zurich, 8032 Zurich, Switzerland.; Center for Applied Biotechnology and Molecular Medicine (CABMM), University of Zurich, 8057 Zurich, Switzerland.
Source: Molecular therapy. Nucleic acids [Mol Ther Nucleic Acids] 2024 May 24; Vol. 35 (3), pp. 102229. Date of Electronic Publication: 2024 May 24 (Print Publication: 2024).
Publication Type: Journal Article
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 101581621 Publication Model: eCollection Cited Medium: Print ISSN: 2162-2531 (Print) Linking ISSN: 21622531 NLM ISO Abbreviation: Mol Ther Nucleic Acids Subsets: PubMed not MEDLINE
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  Data: Targeted knock-in of NCF1 cDNA into the NCF2 locus leads to myeloid phenotypic correction of p47 <superscript>phox</superscript> -deficient chronic granulomatous disease.
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  Data: <searchLink fieldCode="AU" term="%22Siow+KM%22">Siow KM</searchLink>; Division of Gene and Cell Therapy, Institute for Regenerative Medicine, University of Zurich, Schlieren, 8952 Zurich, Switzerland.<br /><searchLink fieldCode="AU" term="%22Güngör+M%22">Güngör M</searchLink>; Division of Gene and Cell Therapy, Institute for Regenerative Medicine, University of Zurich, Schlieren, 8952 Zurich, Switzerland.<br /><searchLink fieldCode="AU" term="%22Wrona+D%22">Wrona D</searchLink>; Division of Gene and Cell Therapy, Institute for Regenerative Medicine, University of Zurich, Schlieren, 8952 Zurich, Switzerland.<br /><searchLink fieldCode="AU" term="%22Raimondi+F%22">Raimondi F</searchLink>; Division of Gene and Cell Therapy, Institute for Regenerative Medicine, University of Zurich, Schlieren, 8952 Zurich, Switzerland.<br /><searchLink fieldCode="AU" term="%22Pastukhov+O%22">Pastukhov O</searchLink>; Division of Gene and Cell Therapy, Institute for Regenerative Medicine, University of Zurich, Schlieren, 8952 Zurich, Switzerland.<br /><searchLink fieldCode="AU" term="%22Tsapogas+P%22">Tsapogas P</searchLink>; Division of Gene and Cell Therapy, Institute for Regenerative Medicine, University of Zurich, Schlieren, 8952 Zurich, Switzerland.<br /><searchLink fieldCode="AU" term="%22Menzi+T%22">Menzi T</searchLink>; Division of Gene and Cell Therapy, Institute for Regenerative Medicine, University of Zurich, Schlieren, 8952 Zurich, Switzerland.<br /><searchLink fieldCode="AU" term="%22Schmitz+M%22">Schmitz M</searchLink>; Department of Biochemistry, University of Zurich, 8057 Zurich, Switzerland.<br /><searchLink fieldCode="AU" term="%22Kulcsár+PI%22">Kulcsár PI</searchLink>; Institute of Pharmacology and Toxicology, University of Zurich, 8057 Zurich, Switzerland.<br /><searchLink fieldCode="AU" term="%22Schwank+G%22">Schwank G</searchLink>; Institute of Pharmacology and Toxicology, University of Zurich, 8057 Zurich, Switzerland.<br /><searchLink fieldCode="AU" term="%22Schulz+A%22">Schulz A</searchLink>; Department of Pediatrics, University Medical Center Ulm, 89075 Ulm, Germany.<br /><searchLink fieldCode="AU" term="%22Jinek+M%22">Jinek M</searchLink>; Department of Biochemistry, University of Zurich, 8057 Zurich, Switzerland.<br /><searchLink fieldCode="AU" term="%22Modlich+U%22">Modlich U</searchLink>; Division of Gene and Cell Therapy, Institute for Regenerative Medicine, University of Zurich, Schlieren, 8952 Zurich, Switzerland.<br /><searchLink fieldCode="AU" term="%22Siler+U%22">Siler U</searchLink>; Division of Gene and Cell Therapy, Institute for Regenerative Medicine, University of Zurich, Schlieren, 8952 Zurich, Switzerland.; School of Life Sciences, Institute for Pharma Technology, University of Applied Sciences and Arts Northwestern Switzerland, 4132 Muttenz, Switzerland.<br /><searchLink fieldCode="AU" term="%22Reichenbach+J%22">Reichenbach J</searchLink>; Division of Gene and Cell Therapy, Institute for Regenerative Medicine, University of Zurich, Schlieren, 8952 Zurich, Switzerland.; Department of Somatic Gene Therapy, University Children's Hospital Zurich, 8032 Zurich, Switzerland.; Center for Applied Biotechnology and Molecular Medicine (CABMM), University of Zurich, 8057 Zurich, Switzerland.
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  Data: <searchLink fieldCode="JN" term="%22101581621%22">Molecular therapy. Nucleic acids</searchLink> [Mol Ther Nucleic Acids] 2024 May 24; Vol. 35 (3), pp. 102229. <i>Date of Electronic Publication: </i>2024 May 24 (<i>Print Publication: </i>2024).
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