From oncogenesis to approval: the lentiviral gene therapy revolution in Wiskott-Aldrich syndrome.
Wiskott-Aldrich syndrome (WAS) is an X-linked primary immunodeficiency caused by pathogenic variants in the WAS gene, resulting in absent or dysfunctional WAS protein (WASp) and a clinical triad of eczema, recurrent infections, and micro-thrombocytopenia. Finally, it explores future innovations, such as advanced precision gene-editing technologies, and discusses strategies to make these life-saving treatments more affordable and accessible on a global scale.
Wiskott-Aldrich syndrome (WAS) is an X-linked primary immunodeficiency caused by pathogenic variants in the WAS gene, resulting in absent or dysfunctional WAS protein (WASp) and a clinical triad of eczema, recurrent infections, and micro-thrombocytopenia. Finally, it explores future innovations, such as advanced precision gene-editing technologies, and discusses strategies to make these life-saving treatments more affordable and accessible on a global scale.
What the study was
- Study design
- Review
- Category
- Drug Development
- Maturity
- Exploratory
- Journal
- Regenerative medicine
Why it surfaced
Score 6/10: Review, human study, Exploratory. ⬜ NONE — Standard addition
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