Article ; Online: Multidimensional Response Surface Methodology for the Development of a Gene Editing Protocol for p67
2024 Volume 35, Issue 7-8, Page(s) 298–312
Abstract: Replacing a faulty gene with a correct copy has become a viable therapeutic option as a result of recent progress in gene editing protocols. Targeted integration of therapeutic genes in hematopoietic stem cells has been achieved for multiple genes using ... ...
Abstract | Replacing a faulty gene with a correct copy has become a viable therapeutic option as a result of recent progress in gene editing protocols. Targeted integration of therapeutic genes in hematopoietic stem cells has been achieved for multiple genes using Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9 system and Adeno-Associated Virus (AAV) to carry a donor template. Although this is a promising strategy to correct genetic blood disorders, it is associated with toxicity and loss of function in CD34 |
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MeSH term(s) | Humans ; Granulomatous Disease, Chronic/genetics ; Granulomatous Disease, Chronic/therapy ; Gene Editing ; Genetic Therapy/methods ; Antigens, CD34/genetics ; Hematopoietic Stem Cells/metabolism ; CRISPR-Cas Systems |
Chemical Substances | Antigens, CD34 |
Language | English |
Publishing date | 2024-01-24 |
Publishing country | United States |
Document type | Journal Article |
ZDB-ID | 1028152-6 |
ISSN | 1557-7422 ; 1043-0342 |
ISSN (online) | 1557-7422 |
ISSN | 1043-0342 |
DOI | 10.1089/hum.2023.114 |
Database | MEDical Literature Analysis and Retrieval System OnLINE |
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