Strategies to treat neurodegeneration in neuronal ceroid lipofuscinosis: a view onto the retina
Author:
Publisher
Medknow
Subject
Developmental Neuroscience
Reference12 articles.
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3. Kleine Holthaus SM, Ribeiro J, Abelleira-Hervas L, Pearson RA, Duran Y, Georgiadis A, Sampson RD, Rizzi M, Hoke J, Maswood R, Azam S, Luhmann UFO, Smith AJ, Mole SE, Ali RR (2018) Prevention of photoreceptor cell loss in a Cln6(nclf) mouse model of Batten disease requires CLN6 gene transfer to bipolar cells. Mol Ther 26:1343-1353.
4. Kleine Holthaus SM, Aristorena M, Maswood R, Semenyuk O, Hoke J, Hare A, Smith AJ, Mole SE, Ali RR (2020) Gene therapy targeting the inner retina rescues the retinal phenotype in a mouse model of CLN3 Batten disease. Hum Gene Ther 31:709-718.
5. Kohlschutter A, Schulz A, Bartsch U, Storch S (2019) Current and emerging treatment strategies for neuronal ceroid lipofuscinoses. CNS Drugs 33:315-325.
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