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. |
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| 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 |
| Database: | MEDLINE Ultimate |
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