Laboratory teams reported locus‑specific editing of the 21‑hydroxylase gene restored adrenal function in a congenital adrenal hyperplasia (CAH) model, illustrating a tangible therapeutic effect from targeted in vivo approaches. The preclinical work demonstrates functional correction of a monogenic endocrine defect using gene‑editing modalities and supports translation toward clinical development. That finding accompanies broader field momentum and scrutiny: reviews and meetings at gene‑therapy forums reinforced both the promise and delivery challenges for in vivo editing, including vector tropism, off‑target risk and payload size constraints. Researchers noted the need for delivery platforms that reliably reach target tissues while limiting systemic exposure. The combined evidence underscores a growing pipeline of in vivo editing strategies aimed at monogenic diseases but highlights manufacturing, regulatory and safety hurdles that developers must clear before broad clinical application. Industry watchers will track whether these early successes accelerate regulatory frameworks and investor support for in‑body editing programs.