Gene Therapy Targeting the Inner Retina Rescues the Retinal Phenotype in a Mouse Model of CLN3 Batten Disease-Reference-Cited by-同舟云学术

Gene Therapy Targeting the Inner Retina Rescues the Retinal Phenotype in a Mouse Model of CLN3 Batten Disease

Published:2020-07-01 Issue:13-14 Volume:31 Page:709-718
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

kleine Holthaus Sophia-Martha¹,Aristorena Mikel¹,Maswood Ryea¹,Semenyuk Olha¹,Hoke Justin¹,Hare Aura¹,Smith Alexander J.¹,Mole Sara E.²³⁴,Ali Robin R.¹⁵

Affiliation:

1. Department of Genetics, UCL Institute of Ophthalmology, London, United Kingdom.

2. MRC Laboratory for Molecular Cell Biology, University College London, London, United Kingdom.

3. UCL Institute of Child Health, London, United Kingdom.

4. UCL Department of Genetics, Evolution and Environment, University College London, London, United Kingdom.

5. NIHR Biomedical Research Centre at Moorfields Eye Hospital NHS Foundation Trust, London, United Kingdom.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2020.038

Reference46 articles.

1. 1. Mole S, Williams R, Goebel H. The Neuronal Ceroid Lipofuscinoses (Batten Disease). Oxford, UK: Oxford University Press, 2011.

2. NCL diseases — clinical perspectives

3. Cell biology and function of neuronal ceroid lipofuscinosis-related proteins

4. Two Motifs Target Batten Disease Protein CLN3 to Lysosomes in Transfected Nonneuronal and Neuronal Cells

5. CLN3 protein is targeted to neuronal synapses but excluded from synaptic vesicles: new clues to Batten disease

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