Life-Limiting Peripheral Organ Dysfunction in Feline Sandhoff Disease Emerges after Effective CNS Gene Therapy.
Johnson AK, McCurdy VJ, Gray-Edwards HL, Maguire AS, Cochran JN, Gross AL, Skinner HE, Randle AN, Shirley JL, Brunson BL, Bradbury AM, Leroy SG, Hwang M, Rockwell HE, Cox NR, Baker HJ, Seyfried TN, Sena-Esteves M, Martin DR.
Johnson AK, et al. Among authors: rockwell he.
Ann Neurol. 2023 Nov;94(5):969-986. doi: 10.1002/ana.26756. Epub 2023 Aug 16.
Ann Neurol. 2023.
PMID: 37526361
Free PMC article.