Gene Editing for ATXN3 Inactivation in Machado-Joseph disease: CRISPR-Cas9 as a Therapeutic Alternative to TALEN-Induced Toxicity
Sara Monteiro Lopes, Miguel Monteiro Lopes, Daniela Camargo de Oliveira, Magda M. Santana, Ana Rita Fernandes, Ana Vasconcelos‐Ferreira, Dina Pereira, Maria Casquinha, Clévio Nóbrega, Carlos A. Matos, Neville E. Sanjana, Patrick D. Hsu, F. Ann Ran, Lukasz J. Swiech, Le Cong, , Luís Pereira de Almeida
This study compares CRISPR-Cas9 and TALEN gene editing in a mouse model of Machado-Joseph disease, showing CRISPR-Cas9 reduces ATXN3 aggregates and delays disease progression.
bioRxiv · Feb 14, 2025 · PreprintNeuroscience