All-in-one gene therapy alternative for DBAS.
basic_science · Level V
Where this comes from
- Record sourced from PubMed, PMID 39753111.
- Also identified by DOI 10.1016/j.stem.2024.12.003.
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Abstract
Diamond-Blackfan anemia syndrome is a ribosomopathy classified among the bone marrow failure syndromes. This disease exhibits significant heterogeneity, with up to 24 genetic variants identified to date. Voit et al.<sup>1</sup> present compelling preclinical evidence supporting a universal lentiviral gene therapy strategy to treat patients, regardless of the specific gene involved.
Medical subject headings
- Genetic Therapy
- Anemia, Diamond-Blackfan