The New Wave of Gene and Cell Therapies Across Diseases - PubMed
5 hours ago
- #gene-therapy
- #cell-therapy
- #CRISPR
- Rapid progress in gene and cell therapies for genetic diseases and conditions like type 1 diabetes.
- Technological advances in CAR-T and CAR-NK cells (using CRISPR or transgenes) have improved cancer therapy.
- Emergence of 'off-the-shelf' cellular therapies to mitigate alloreactivity and immune rejection.
- Different diseases require tailored treatment strategies due to distinct biological and logistical limitations.
- Modification of therapeutic cells ex vivo or in vivo using LNPs, AAV vectors, and lentiviral vectors.
- Focus on selecting suitable promoters for specific expression in targeted cell types.
- Applications in treating cancer, hereditary diseases (e.g., Huntington's, CPS1 deficiency), and emerging uses in heart diseases and diabetes.
- Therapeutic strategies include tRNA gene editing.
- Authors highlight the most representative, practical, and up-to-date examples of therapies.