Toward a translational gene therapy for mucolipidosis IV by Brina Snyder, Rachel M. Bailey

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Editorial: Personalized medicine for neuromuscular disorders by Marc Bartoli, Rachel M. Bailey, Rachel M. Bailey, Kathrin Meyer, Kathrin Meyer, Florian Barthélémy, Florian Barthélémy

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Gene Therapy: Novel Approaches to Targeting Monogenic Epilepsies by Kimberly Goodspeed, Rachel M. Bailey, Rachel M. Bailey, Suyash Prasad, Chanchal Sadhu, Jessica A. Cardenas, Mary Holmay, Deborah A. Bilder, Berge A. Minassian

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Development of Intrathecal AAV9 Gene Therapy for Giant Axonal Neuropathy by Rachel M. Bailey, Diane Armao, Sahana Nagabhushan Kalburgi, Steven J. Gray

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Advancing the pathologic phenotype of giant axonal neuropathy: early involvement of the ocular lens by Diane Armao, Thomas W. Bouldin, Rachel M. Bailey, Jody E. Hooper, Diana X. Bharucha, Steven J. Gray

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Preclinical use of a clinically-relevant scAAV9/SUMF1 vector for the treatment of multiple sulfatase deficiency by Maximiliano Presa, Rachel M. Bailey, Somdatta Ray, Lauren Bailey, Saurabh Tata, Tara Murphy, Pierre-Alexandre Piec, Harold Combs, Steven J. Gray, Cathleen Lutz

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