Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis by CNS Administration of a Serotype 2 Adeno-Associated Virus Expressing CLN2 cDNA-Reference-Cited by-同舟云学术

Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis by CNS Administration of a Serotype 2 Adeno-Associated Virus Expressing CLN2 cDNA

Published:2008-05 Issue:5 Volume:19 Page:463-474
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Worgall Stefan¹²,Sondhi Dolan¹,Hackett Neil R.¹,Kosofsky Barry²,Kekatpure Minal V.²,Neyzi Nurunisa²,Dyke Jonathan P.³,Ballon Douglas³,Heier Linda³,Greenwald Bruce M.²,Christos Paul⁴,Mazumdar Madhu⁴,Souweidane Mark M.⁵,Kaplitt Michael G.⁵,Crystal Ronald G.¹

Affiliation:

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

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

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

4. Department of Public Health, Weill Cornell Medical College, New York, NY 10065.

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

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2008.022