Slowing late infantile Batten disease by direct brain parenchymal administration of a rh.10 adeno-associated virus expressing CLN2-Reference-Cited by-同舟云学术

Slowing late infantile Batten disease by direct brain parenchymal administration of a rh.10 adeno-associated virus expressing CLN2

Published:2020-12-02 Issue:572 Volume:12 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Sondhi Dolan¹^ORCID,Kaminsky Stephen M.¹,Hackett Neil R.¹,Pagovich Odelya E.¹,Rosenberg Jonathan B.¹,De Bishnu P.¹,Chen Alvin¹,Van de Graaf Benjamin¹,Mezey Jason G.¹²,Mammen Grace W.¹,Mancenido Denesy¹,Xu Fang¹,Kosofsky Barry³,Yohay Kaleb³^ORCID,Worgall Stefan¹³^ORCID,Kaner Robert J.¹⁴^ORCID,Souwedaine Mark⁵^ORCID,Greenwald Bruce M.³,Kaplitt Michael⁵^ORCID,Dyke Jonathan P.⁶^ORCID,Ballon Douglas J.¹⁶^ORCID,Heier Linda A.⁶^ORCID,Kiss Szilard⁷^ORCID,Crystal Ronald G.¹⁴^ORCID

Affiliation:

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

2. Department of Biological Statistics and Computational Biology, Cornell University, Ithaca, NY 14853, USA.

3. Department of Pediatrics, Weill Cornell Medical College, New York, NY 10065, USA.

4. Department of Medicine, Weill Cornell Medical College, New York, NY 10065, USA.

5. Department of Neurological Surgery, Weill Cornell Medical College, New York, NY 10065, USA.

6. Department of Radiology, Weill Cornell Medical College, New York, NY 10065, USA.

7. Department of Ophthalmology, Retina Service, Weill Cornell Medical College, New York, NY 10065, USA.

Abstract

Administration of an adeno-associated virus coding for the CLN2 gene into the brain parenchyma slows the progression of CLN2 disease in children.

Funder

National Institutes of Health

National Institute of Neurological Disorders and Stroke

Nathan’s Battle

Cures Within Reach

Noah’s Hope

Hope4Bridget

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

Reference68 articles.

1. Association of Mutations in a Lysosomal Protein with Classical Late-Infantile Neuronal Ceroid Lipofuscinosis

2. R. E. Williams I. Gottlob B. D. Lake H. H. Goebel W. Wheeler R. B. Wheeler in The Neuronal Ceroid Lipofuscinoses (Batten Disease) H. H. Goebel S. E. Mole B. D. Lake Eds. (IOS Press 1999) pp. 37–54.

3. The Neuronal Ceroid-Lipofuscinoses

4. Neurological deterioration in late infantile neuronal ceroid lipofuscinosis

5. Disease characteristics and progression in patients with late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease: an observational cohort study

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