Gene correction for SCID-X1 in long-term hematopoietic stem cells
Gene correction in hematopoietic stem cells could be a powerful way to treat monogenic diseases of the blood and immune system. Here the authors develop a strategy using CRISPR-Cas9 and an aAdeno-Associated vVirus(AAV)-delivered IL2RG cDNA to correct X-linked sSevere Ccombined iImmunodeficiency (SCI...
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| Format: | Article |
| Language: | English |
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Nature Publishing Group
2019-04-01
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| Series: | Nature Communications |
| Online Access: | https://doi.org/10.1038/s41467-019-09614-y |
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doaj-66cf921ac2ec4178bebe47ca77ff7c87 |
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doaj-66cf921ac2ec4178bebe47ca77ff7c872021-05-11T11:56:55ZengNature Publishing GroupNature Communications2041-17232019-04-0110111510.1038/s41467-019-09614-yGene correction for SCID-X1 in long-term hematopoietic stem cellsMara Pavel-Dinu0Volker Wiebking1Beruh T. Dejene2Waracharee Srifa3Sruthi Mantri4Carmencita E. Nicolas5Ciaran Lee6Gang Bao7Eric J. Kildebeck8Niraj Punjya9Camille Sindhu10Matthew A. Inlay11Nivedita Saxena12Suk See DeRavin13Harry Malech14Maria Grazia Roncarolo15Kenneth I. Weinberg16Matthew H. Porteus17Department of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine, Stanford UniversityDepartment of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine, Stanford UniversityDepartment of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine, Stanford UniversityDepartment of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine, Stanford UniversityDepartment of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine, Stanford UniversityDepartment of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine, Stanford UniversityDepartment of Bioengineering, Rice UniversityDepartment of Bioengineering, Rice UniversityCenter for Engineering Innovation, University of Texas at DallasDepartment of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine, Stanford UniversityDepartment of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine, Stanford UniversityDepartment of Cellular and Molecular Biosciences, University of California IrvineDepartment of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine, Stanford UniversityLaboratory of Host Defenses, National Institutes of Allergy and Infectious Diseases, National Institute of HealthLaboratory of Host Defenses, National Institutes of Allergy and Infectious Diseases, National Institute of HealthDepartment of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine, Stanford UniversityDepartment of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine, Stanford UniversityDepartment of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine, Stanford UniversityGene correction in hematopoietic stem cells could be a powerful way to treat monogenic diseases of the blood and immune system. Here the authors develop a strategy using CRISPR-Cas9 and an aAdeno-Associated vVirus(AAV)-delivered IL2RG cDNA to correct X-linked sSevere Ccombined iImmunodeficiency (SCID-X1) with a high success rate.https://doi.org/10.1038/s41467-019-09614-y |
| collection |
DOAJ |
| language |
English |
| format |
Article |
| sources |
DOAJ |
| author |
Mara Pavel-Dinu Volker Wiebking Beruh T. Dejene Waracharee Srifa Sruthi Mantri Carmencita E. Nicolas Ciaran Lee Gang Bao Eric J. Kildebeck Niraj Punjya Camille Sindhu Matthew A. Inlay Nivedita Saxena Suk See DeRavin Harry Malech Maria Grazia Roncarolo Kenneth I. Weinberg Matthew H. Porteus |
| spellingShingle |
Mara Pavel-Dinu Volker Wiebking Beruh T. Dejene Waracharee Srifa Sruthi Mantri Carmencita E. Nicolas Ciaran Lee Gang Bao Eric J. Kildebeck Niraj Punjya Camille Sindhu Matthew A. Inlay Nivedita Saxena Suk See DeRavin Harry Malech Maria Grazia Roncarolo Kenneth I. Weinberg Matthew H. Porteus Gene correction for SCID-X1 in long-term hematopoietic stem cells Nature Communications |
| author_facet |
Mara Pavel-Dinu Volker Wiebking Beruh T. Dejene Waracharee Srifa Sruthi Mantri Carmencita E. Nicolas Ciaran Lee Gang Bao Eric J. Kildebeck Niraj Punjya Camille Sindhu Matthew A. Inlay Nivedita Saxena Suk See DeRavin Harry Malech Maria Grazia Roncarolo Kenneth I. Weinberg Matthew H. Porteus |
| author_sort |
Mara Pavel-Dinu |
| title |
Gene correction for SCID-X1 in long-term hematopoietic stem cells |
| title_short |
Gene correction for SCID-X1 in long-term hematopoietic stem cells |
| title_full |
Gene correction for SCID-X1 in long-term hematopoietic stem cells |
| title_fullStr |
Gene correction for SCID-X1 in long-term hematopoietic stem cells |
| title_full_unstemmed |
Gene correction for SCID-X1 in long-term hematopoietic stem cells |
| title_sort |
gene correction for scid-x1 in long-term hematopoietic stem cells |
| publisher |
Nature Publishing Group |
| series |
Nature Communications |
| issn |
2041-1723 |
| publishDate |
2019-04-01 |
| description |
Gene correction in hematopoietic stem cells could be a powerful way to treat monogenic diseases of the blood and immune system. Here the authors develop a strategy using CRISPR-Cas9 and an aAdeno-Associated vVirus(AAV)-delivered IL2RG cDNA to correct X-linked sSevere Ccombined iImmunodeficiency (SCID-X1) with a high success rate. |
| url |
https://doi.org/10.1038/s41467-019-09614-y |
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