Sophia-martha Holthaus-Kleine
London, ENG, United Kingdom
Last Updated: 09/26/2026
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Found 8 publications
Experimental gene therapies for the NCLs.
Journal: Biochimica et biophysica acta. Molecular basis of disease
Published: November 06, 2019
Neonatal brain-directed gene therapy rescues a mouse model of neurodegenerative CLN6 Batten disease.
Neonatal brain-directed gene therapy rescues a mouse model of neurodegenerative CLN6 Batten disease.
Journal: Human molecular genetics
Published: June 12, 2019
Gene Therapy Approaches to Treat the Neurodegeneration and Visual Failure in Neuronal Ceroid Lipofuscinoses.
Journal: Advances in experimental medicine and biology
Published: May 04, 2018
Clinical challenges and future therapeutic approaches for neuronal ceroid lipofuscinosis.
Journal: The Lancet. Neurology
Published: February 19, 2018
Prevention of Photoreceptor Cell Loss in a Cln6nclf Mouse Model of Batten Disease Requires CLN6 Gene Transfer to Bipolar Cells.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: November 27, 2017
Multimodal analysis of ocular inflammation using the endotoxin-induced uveitis mouse model.
Journal: Disease models & mechanisms
Published: September 03, 2015
Showing 1-8 of 8
Last Updated: 09/26/2026