CRISPR-Cas9 in hiPSCs: A new era in personalized treatment for Stargardt disease

Inherited retinal dystrophies, including Stargardt disease, are a group of genetic eye diseases that currently lack effective treatment options. The CRISPR-Cas9 gene editing system and human induced pluripotent stem cells (hiPSCs) offer a promising avenue for treating Stargardt disease, a devastating genetic eye disorder. In a recent study published in Molecular Therapy–Nucleic Acids, Siles and colleagues demonstrated the accurate correction of two pathogenic variants in the ABCA4 gene, which triggers Stargardt disease, in hiPSCs without any adverse effects.