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Gene therapy involves the introduction of new genes into cells, to restore or add gene expression, for the purpose of treating disease. Most commonly a mutated gene is replaced with DNA encoding a functional copy. Alternatively DNA encoding a therapeutic protein drug may be introduced.
A therapeutic strategy that alters gene expression in a rare and severe neurodevelopmental condition has been tested in stem-cell-based models of the disease, and has been shown to correct genetic and cellular defects.
Precise genome editing is crucial. Here the authors demonstrate that dual inhibition of p53-mediated cell death and distinct activation of the DNA damage repair system upon DNA damage by cytosine base editor (CBE) or prime editor (PE) additively enhanced editing efficiency in hPSCs.
Bietti crystalline corneoretinal dystrophy (BCD) is an autosomal recessive chorioretinal degenerative disease without approved therapeutic drug. Here, the authors show a promising CRISPR/Cas9 mediated homology-independent targeted integration therapy in patient derived cells and humanized mice carrying BCD mutations.
In non-human primates, rAAVs are delivered through suprachoroidal injection at a high dose to achieve optimal efficacy. Here, the authors present a novel AAV capsid (AAVv128) that significantly improved the transduction efficiency in photoreceptor and retinal pigment epithelial cells across species.
Final results of two studies — whose preliminary data led to regulatory approval of the gene therapy exagamglogene autotemcel — describe highly effective treatment of sickle-cell disease and transfusion-dependent β-thalassemia.
A therapeutic strategy that alters gene expression in a rare and severe neurodevelopmental condition has been tested in stem-cell-based models of the disease, and has been shown to correct genetic and cellular defects.
A new approach to modify the epigenome can lead to durable silencing of Pcsk9 in mice, thereby reducing plasma LDL-cholesterol levels, according to a study published in Nature.