A Health Impact Assessment of CRISPR-Cas9 as a Therapeutic Approach for Prader-Willi Syndrome
DOI:
https://doi.org/10.36877/pddbs.a0000592Abstract
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.
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Copyright (c) 2025 Rachel Xue-Yu Teoh, Nancy Choon-Si Ng, Rebecca Shin-Yee Wong
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