Partial Correction of the CNS Lysosomal Storage Defect in a Mouse Model of Juvenile Neuronal Ceroid Lipofuscinosis by Neonatal CNS Administration of an Adeno-Associated Virus Serotype rh.10 Vector Expressing the Human CLN3 Gene-Reference-Cited by-同舟云学术

Partial Correction of the CNS Lysosomal Storage Defect in a Mouse Model of Juvenile Neuronal Ceroid Lipofuscinosis by Neonatal CNS Administration of an Adeno-Associated Virus Serotype rh.10 Vector Expressing the Human CLN3 Gene

Published:2014-03 Issue:3 Volume:25 Page:223-239
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Sondhi Dolan¹,Scott Emma C.¹,Chen Alvin¹,Hackett Neil R.¹,Wong Andrew M.S.²,Kubiak Agnieszka²,Nelvagal Hemanth R.²,Pearse Yewande²,Cotman Susan L.³,Cooper Jonathan D.²,Crystal Ronald G.¹

Affiliation:

1. Department of Genetic Medicine, Weill Medical College, Cornell University, New York, NY 10065.

2. Department of Neuroscience, Centre for the Cellular Basis of Behaviour, and King's Health Partners Centre for Neurodegeneration Research, The Institute of Psychiatry, King's College London, London SE5 9NU, United Kingdom.

3. Center for Human Genetic Research, Massachusetts General Hospital, Boston, MA 02114.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2012.253

Reference62 articles.

1. Protracted juvenile neuronal ceroid lipofuscinosis—An autopsy report and immunohistochemical analysis

2. MRI of neuronal ceroid lipofuscinosis. II. Postmortem MRI and histopathological study of the brain in 16 cases of neuronal ceroid lipofuscinosis of juvenile or late infantile type

3. Regional and cellular neuropathology in the palmitoyl protein thioesterase-1 null mutant mouse model of infantile neuronal ceroid lipofuscinosis

4. Genetic therapy for the nervous system

5. Neuronal ceroid lipofuscinosis (nclf), a new disorder of the mouse linked to chromosome 9

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