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Advancing gene targeting for primary immune deficiencies: Adenine base editing of the human IL2RG locus for correction of SCID-X1.
- Source :
-
Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2024 Jun 05; Vol. 32 (6), pp. 1606-1608. Date of Electronic Publication: 2024 May 22. - Publication Year :
- 2024
-
Abstract
- Competing Interests: Declaration of interests R.S.M. is an employee of and has equity in Immusoft Corporation.
- Subjects :
- Humans
Gene Targeting
Genetic Therapy methods
Adenine analogs & derivatives
Animals
CRISPR-Cas Systems
Interleukin Receptor Common gamma Subunit genetics
Interleukin Receptor Common gamma Subunit deficiency
Gene Editing methods
X-Linked Combined Immunodeficiency Diseases genetics
X-Linked Combined Immunodeficiency Diseases therapy
Subjects
Details
- Language :
- English
- ISSN :
- 1525-0024
- Volume :
- 32
- Issue :
- 6
- Database :
- MEDLINE
- Journal :
- Molecular therapy : the journal of the American Society of Gene Therapy
- Publication Type :
- Academic Journal
- Accession number :
- 38781958
- Full Text :
- https://doi.org/10.1016/j.ymthe.2024.05.026