Assessment of Safety and Biodistribution of AAVrh.10hCLN2 Following Intracisternal Administration in Nonhuman Primates for the Treatment of CLN2 Batten Disease-Reference-Cited by-同舟云学术

Assessment of Safety and Biodistribution of AAVrh.10hCLN2 Following Intracisternal Administration in Nonhuman Primates for the Treatment of CLN2 Batten Disease

Published:2023-09-01 Issue:17-18 Volume:34 Page:905-916
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

De Bishnu P.¹,Rosenberg Jonathan B.¹,Selvan Nithya²,Wilson Isabelle¹,Yusufzai Nadir¹,Greco Alessandria¹,Kaminsky Stephen M.¹^ORCID,Heier Linda A.³,Ricart Arbona Rodolfo J.⁴,Miranda Ileana C.⁵^ORCID,Monette Sebastien⁵,Nair Anju²,Khanna Richie²,Crystal Ronald G.¹^ORCID,Sondhi Dolan¹^ORCID

Affiliation:

1. Department of Genetic Medicine, New York, New York, USA

2. LEXEO Therapeutics, New York, New York, USA.

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

4. Center for Comparative Medicine and Pathology, Memorial Sloan Kettering Cancer Center, Weill Cornell Medicine, New York, New York, USA.

5. Laboratory of Comparative Pathology, Memorial Sloan Kettering Cancer Center, The Rockefeller University, Weill Cornell Medicine, New York, New York, USA.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2023.067

Reference51 articles.

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2. 2. Williams RE, Gottlob I, Lake BD, et al. CLN2 classic late infantile NCL. In: The Neuronal Ceroid Lipofuscinoses (Batten Disease). (Goebel HH, Mole SE, Lake BD. eds.) IOS Press: Amsterdam; 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