CRISPR/Cas-Based Gene Editing Strategies for DOCK8 Immunodeficiency Syndrome
Defects in the DOCK8 gene causes combined immunodeficiency termed DOCK8 immunodeficiency syndrome (DIDS). DIDS previously belonged to the disease category of autosomal recessive hyper IgE syndrome (AR-HIES) but is now classified as a combined immunodeficiency (CID). This genetic disorder induces ear...
| Main Authors: | , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Frontiers Media S.A.
2022-03-01
|
| Series: | Frontiers in Genome Editing |
| Subjects: | |
| Online Access: | https://www.frontiersin.org/articles/10.3389/fgeed.2022.793010/full |
| _version_ | 1818992017103912960 |
|---|---|
| author | Sujan Ravendran Sabina Sánchez Hernández Saskia König Rasmus O. Bak |
| author_facet | Sujan Ravendran Sabina Sánchez Hernández Saskia König Rasmus O. Bak |
| author_sort | Sujan Ravendran |
| collection | DOAJ |
| description | Defects in the DOCK8 gene causes combined immunodeficiency termed DOCK8 immunodeficiency syndrome (DIDS). DIDS previously belonged to the disease category of autosomal recessive hyper IgE syndrome (AR-HIES) but is now classified as a combined immunodeficiency (CID). This genetic disorder induces early onset of susceptibility to severe recurrent viral and bacterial infections, atopic diseases and malignancy resulting in high morbidity and mortality. This pathological state arises from impairment of actin polymerization and cytoskeletal rearrangement, which induces improper immune cell migration-, survival-, and effector functions. Owing to the severity of the disease, early allogenic hematopoietic stem cell transplantation is recommended even though it is associated with risk of unintended adverse effects, the need for compatible donors, and high expenses. So far, no alternative therapies have been developed, but the monogenic recessive nature of the disease suggests that gene therapy may be applied. The advent of the CRISPR/Cas gene editing system heralds a new era of possibilities in precision gene therapy, and positive results from clinical trials have already suggested that the tool may provide definitive cures for several genetic disorders. Here, we discuss the potential application of different CRISPR/Cas-mediated genetic therapies to correct the DOCK8 gene. Our findings encourage the pursuit of CRISPR/Cas-based gene editing approaches, which may constitute more precise, affordable, and low-risk definitive treatment options for DOCK8 deficiency. |
| first_indexed | 2024-12-20T20:19:28Z |
| format | Article |
| id | doaj.art-c2ee6c3100f54fa1b3548f0898727f10 |
| institution | Directory Open Access Journal |
| issn | 2673-3439 |
| language | English |
| last_indexed | 2024-12-20T20:19:28Z |
| publishDate | 2022-03-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Genome Editing |
| spelling | doaj.art-c2ee6c3100f54fa1b3548f0898727f102022-12-21T19:27:36ZengFrontiers Media S.A.Frontiers in Genome Editing2673-34392022-03-01410.3389/fgeed.2022.793010793010CRISPR/Cas-Based Gene Editing Strategies for DOCK8 Immunodeficiency SyndromeSujan RavendranSabina Sánchez HernándezSaskia KönigRasmus O. BakDefects in the DOCK8 gene causes combined immunodeficiency termed DOCK8 immunodeficiency syndrome (DIDS). DIDS previously belonged to the disease category of autosomal recessive hyper IgE syndrome (AR-HIES) but is now classified as a combined immunodeficiency (CID). This genetic disorder induces early onset of susceptibility to severe recurrent viral and bacterial infections, atopic diseases and malignancy resulting in high morbidity and mortality. This pathological state arises from impairment of actin polymerization and cytoskeletal rearrangement, which induces improper immune cell migration-, survival-, and effector functions. Owing to the severity of the disease, early allogenic hematopoietic stem cell transplantation is recommended even though it is associated with risk of unintended adverse effects, the need for compatible donors, and high expenses. So far, no alternative therapies have been developed, but the monogenic recessive nature of the disease suggests that gene therapy may be applied. The advent of the CRISPR/Cas gene editing system heralds a new era of possibilities in precision gene therapy, and positive results from clinical trials have already suggested that the tool may provide definitive cures for several genetic disorders. Here, we discuss the potential application of different CRISPR/Cas-mediated genetic therapies to correct the DOCK8 gene. Our findings encourage the pursuit of CRISPR/Cas-based gene editing approaches, which may constitute more precise, affordable, and low-risk definitive treatment options for DOCK8 deficiency.https://www.frontiersin.org/articles/10.3389/fgeed.2022.793010/fullgene editing (CRISPR-Cas9)CRISPR/Cas 9hematopoietic stem cellDOCK8 immunodeficiency syndromeDOCK8 deficiencyDOCK8 |
| spellingShingle | Sujan Ravendran Sabina Sánchez Hernández Saskia König Rasmus O. Bak CRISPR/Cas-Based Gene Editing Strategies for DOCK8 Immunodeficiency Syndrome Frontiers in Genome Editing gene editing (CRISPR-Cas9) CRISPR/Cas 9 hematopoietic stem cell DOCK8 immunodeficiency syndrome DOCK8 deficiency DOCK8 |
| title | CRISPR/Cas-Based Gene Editing Strategies for DOCK8 Immunodeficiency Syndrome |
| title_full | CRISPR/Cas-Based Gene Editing Strategies for DOCK8 Immunodeficiency Syndrome |
| title_fullStr | CRISPR/Cas-Based Gene Editing Strategies for DOCK8 Immunodeficiency Syndrome |
| title_full_unstemmed | CRISPR/Cas-Based Gene Editing Strategies for DOCK8 Immunodeficiency Syndrome |
| title_short | CRISPR/Cas-Based Gene Editing Strategies for DOCK8 Immunodeficiency Syndrome |
| title_sort | crispr cas based gene editing strategies for dock8 immunodeficiency syndrome |
| topic | gene editing (CRISPR-Cas9) CRISPR/Cas 9 hematopoietic stem cell DOCK8 immunodeficiency syndrome DOCK8 deficiency DOCK8 |
| url | https://www.frontiersin.org/articles/10.3389/fgeed.2022.793010/full |
| work_keys_str_mv | AT sujanravendran crisprcasbasedgeneeditingstrategiesfordock8immunodeficiencysyndrome AT sabinasanchezhernandez crisprcasbasedgeneeditingstrategiesfordock8immunodeficiencysyndrome AT saskiakonig crisprcasbasedgeneeditingstrategiesfordock8immunodeficiencysyndrome AT rasmusobak crisprcasbasedgeneeditingstrategiesfordock8immunodeficiencysyndrome |