A Health Impact Assessment of CRISPR-Cas9 as a Therapeutic Approach for Prader-Willi Syndrome

Rachel Xue-Yu Teoh, Nancy Choon-Si Ng, Rebecca Shin-Yee Wong — Sun, 04 May 2025 00:00:00 +0800

Prader-Willi Syndrome is a rare neurodevelopmental genetic disorder resulting from expression defects of the exclusively paternally-expressed PWS locus at chromosome 15q11-q13. Even though its multifaceted manifestations and potentially fatal comorbidities poses universal concern, it remains incurable. Thus, this commentary applies a health impact assessment framework to evaluate the potential behind CRISPR-Cas9 gene-editing technology as a therapeutic intervention for PWS. While this novel technique displays outstanding precision and effectiveness in the targeted identification and demethylation of the silenced maternal locus, challenges such as off-target mutations, safety, and ethical implications warrant additional improvements. Therefore, recommendations regarding future research and clinical implementation are also proposed.

Progress in Drug Discovery & Biomedical Science

A Health Impact Assessment of CRISPR-Cas9 as a Therapeutic Approach for Prader-Willi Syndrome