Cross-species efficacy of enzyme replacement therapy for CLN1 disease in mice and sheep-Reference-Cited by-同舟云学术

Cross-species efficacy of enzyme replacement therapy for CLN1 disease in mice and sheep

Published:2022-10-17 Issue:20 Volume:132 Page:
ISSN:1558-8238
Container-title:Journal of Clinical Investigation
language:en
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Author:

Nelvagal Hemanth R.^ORCID,Eaton Samantha L.,Wang Sophie H.,Eultgen Elizabeth M.,Takahashi Keigo^ORCID,Le Steven Q.,Nesbitt Rachel,Dearborn Joshua T.^ORCID,Siano Nicholas,Puhl Ana C.^ORCID,Dickson Patricia I.,Thompson Gerard,Murdoch Fraser,Brennan Paul M.^ORCID,Gray Mark^ORCID,Greenhalgh Stephen N.^ORCID,Tennant Peter,Gregson Rachael^ORCID,Clutton Eddie^ORCID,Nixon James,Proudfoot Chris^ORCID,Guido Stefano^ORCID,Lillico Simon G.,Whitelaw C. Bruce A.^ORCID,Lu Jui-Yun,Hofmann Sandra L.^ORCID,Ekins Sean,Sands Mark S.,Wishart Thomas M.^ORCID,Cooper Jonathan D.^ORCID

Funder

NINDS National Institutes of Health

Hailey’s Heroes

Biotechnology and Biological Sciences Research Council

Department of Pediatrics, Washington University in St Louis, School of Medicine

McDonnell International Scholars Academy

The RS Macdonald Charitable Trust

Publisher

American Society for Clinical Investigation

Subject

General Medicine

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