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Molecular Therapy : the Journal of the American Society of Gene Therapy|July 19, 2025
AAV-delivered PPT1 provides long-term neurological benefits in CLN1 mice and achieves therapeutic levels in sheep brainMd Suhail Alam, Apeksha Khatiwada, Samantha L Eaton, et al.
The Journal of Clinical Investigation|August 30, 2022
Cross-species efficacy of enzyme replacement therapy for CLN1 disease in mice and sheepHemanth R Nelvagal, Samantha L Eaton, Sophie H Wang, et al.
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