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Assessment of AAV Vector Tropisms for Mouse and Human Pluripotent Stem Cell-Derived RPE and Photoreceptor Cells.
Hum Gene Ther. 2018 Oct;29(10):1124-1139. doi: 10.1089/hum.2018.027. Epub 2018 May 14.
Hum Gene Ther. 2018.
PMID: 29580100
Prevention of Photoreceptor Cell Loss in a Cln6nclf Mouse Model of Batten Disease Requires CLN6 Gene Transfer to Bipolar Cells.
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.
Kleine Holthaus SM, et al.
Mol Ther. 2018 May 2;26(5):1343-1353. doi: 10.1016/j.ymthe.2018.02.027. Epub 2018 Mar 2.
Mol Ther. 2018.
PMID: 29606505
Free PMC article.
Item in Clipboard
Gene Therapy Targeting the Inner Retina Rescues the Retinal Phenotype in a Mouse Model of CLN3 Batten Disease.
Kleine Holthaus SM, Aristorena M, Maswood R, Semenyuk O, Hoke J, Hare A, Smith AJ, Mole SE, Ali RR.
Kleine Holthaus SM, et al.
Hum Gene Ther. 2020 Jul;31(13-14):709-718. doi: 10.1089/hum.2020.038.
Hum Gene Ther. 2020.
PMID: 32578444
Free PMC article.
Item in Clipboard
Gene Therapy Approaches to Treat the Neurodegeneration and Visual Failure in Neuronal Ceroid Lipofuscinoses.
Kleine Holthaus SM, Smith AJ, Mole SE, Ali RR.
Kleine Holthaus SM, et al.
Adv Exp Med Biol. 2018;1074:91-99. doi: 10.1007/978-3-319-75402-4_12.
Adv Exp Med Biol. 2018.
PMID: 29721932
Review.
Item in Clipboard
Neonatal brain-directed gene therapy rescues a mouse model of neurodegenerative CLN6 Batten disease.
Kleine Holthaus SM, Herranz-Martin S, Massaro G, Aristorena M, Hoke J, Hughes MP, Maswood R, Semenyuk O, Basche M, Shah AZ, Klaska IP, Smith AJ, Mole SE, Rahim AA, Ali RR.
Kleine Holthaus SM, et al.
Hum Mol Genet. 2019 Dec 1;28(23):3867-3879. doi: 10.1093/hmg/ddz210.
Hum Mol Genet. 2019.
PMID: 31807779
Item in Clipboard
Multimodal analysis of ocular inflammation using the endotoxin-induced uveitis mouse model.
Chu CJ, Gardner PJ, Copland DA, Liyanage SE, Gonzalez-Cordero A, Kleine Holthaus SM, Luhmann UF, Smith AJ, Ali RR, Dick AD.
Chu CJ, et al. Among authors: kleine holthaus sm.
Dis Model Mech. 2016 Apr;9(4):473-81. doi: 10.1242/dmm.022475. Epub 2016 Jan 21.
Dis Model Mech. 2016.
PMID: 26794131
Free PMC article.
Item in Clipboard
Experimental gene therapies for the NCLs.
Liu W, Kleine-Holthaus SM, Herranz-Martin S, Aristorena M, Mole SE, Smith AJ, Ali RR, Rahim AA.
Liu W, et al. Among authors: kleine holthaus sm.
Biochim Biophys Acta Mol Basis Dis. 2020 Sep 1;1866(9):165772. doi: 10.1016/j.bbadis.2020.165772. Epub 2020 Mar 24.
Biochim Biophys Acta Mol Basis Dis. 2020.
PMID: 32220628
Free article.
Review.
Item in Clipboard
Clinical challenges and future therapeutic approaches for neuronal ceroid lipofuscinosis.
Mole SE, Anderson G, Band HA, Berkovic SF, Cooper JD, Kleine Holthaus SM, McKay TR, Medina DL, Rahim AA, Schulz A, Smith AJ.
Mole SE, et al. Among authors: kleine holthaus sm.
Lancet Neurol. 2019 Jan;18(1):107-116. doi: 10.1016/S1474-4422(18)30368-5. Epub 2018 Nov 21.
Lancet Neurol. 2019.
PMID: 30470609
Review.
Item in Clipboard
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