| _version_ | 1866902190036090880 |
|---|---|
| author | Fuertes Oliva, Martín |
| author_facet | Fuertes Oliva, Martín |
| contents | <p>Down syndrome (DS) is the most prevalent chromosomal condition associated with intellectual disability, primarily caused by a trisomy of chromosome 21. Despite significant clinical support and early intervention strategies, no effective treatment exists that targets the root genomic causes of DS. This article proposes a modular, reversible, and personalized CRISPR-based therapeutic strategy involving epigenetic silencing of the extra chromosome 21, repression of key overexpressed genes, and enhancement of neuroprotective gene expression. The modular design enables customization based on individual genetic and transcriptomic profiles.</p> |
| format | Recurso digital |
| id | zenodo_https___doi_org_10_5281_zenodo_15450539 |
| institution | Zenodo |
| language | |
| publishDate | 2025 |
| publisher | Zenodo |
| record_format | zenodo |
| spellingShingle | Modular CRISPR-Based Therapeutic Design for Down Syndrome: A Reversible and Personalized Approach Fuertes Oliva, Martín <p>Down syndrome (DS) is the most prevalent chromosomal condition associated with intellectual disability, primarily caused by a trisomy of chromosome 21. Despite significant clinical support and early intervention strategies, no effective treatment exists that targets the root genomic causes of DS. This article proposes a modular, reversible, and personalized CRISPR-based therapeutic strategy involving epigenetic silencing of the extra chromosome 21, repression of key overexpressed genes, and enhancement of neuroprotective gene expression. The modular design enables customization based on individual genetic and transcriptomic profiles.</p> |
| title | Modular CRISPR-Based Therapeutic Design for Down Syndrome: A Reversible and Personalized Approach |
| url | https://doi.org/10.5281/zenodo.15450539 |