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CRISPR-Cas9 guided RNA-based model for the silencing of spinal bulbar muscular atrophy: A functional genetic disorder.

Cellular and molecular biology (Noisy-le-Grand, France) - 8 Oct 2024

Naveed Muhammad, Tabassum Natasha, Aziz Tariq, Aqib Shabbir Muhammad, Alkhateeb Mariam Abdulaziz, Alghamdi Saad, Babalghith Ahmad O, Alsaiari Ahad Amer, Alshareef Sahar A, Barqawi Aminah A

Abstract excerpt

This study explores a novel therapeutic approach for spinal bulbar muscular atrophy (SBMA), a neurodegenerative disorder caused by a mutation in the Androgen Receptor (AR) gene. The aim is to investigate the potential of CRISPR-Cas9 technology in targeting the mutant AR gene to inhibit its production. The objectives include assessing the accuracy and efficacy of CRISPR-Cas9 guided RNAs in silencing the mutant...

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PMID: 39380278
DOI: 10.14715/cmb/2024.70.9.10

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