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480. Gene Therapy for Spinal Muscular Atrophy Type 1 Shows Potential to Improve Survival and Motor Functional Outcomes

Title: 480. Gene Therapy for Spinal Muscular Atrophy Type 1 Shows Potential to Improve Survival and Motor Functional Outcomes
Authors: Mendell, Jerry R.; Al-Zaidy, Samiah; Shell, Richard; Arnold, W Dave; Rodino-Klapac, Louise; Kissel, John T.; Prior, Thomas W.; Miranda, Carlos; Lowes, Linda; Alfano, Lindsay; Berry, Katherine; Petek, Christopher; Church, Kathleen; Braun, Lyndsey; Corcoran, Sarah; Meyer, Kathrin; Likhite, Shibi; Burghes, Arthur HM; Foust, Kevin D.; Kaspar, Brian K.
Source: Molecular Therapy ; volume 24, page S190 ; ISSN 1525-0016
Publisher Information: Elsevier BV
Publication Year: 2016
Collection: ScienceDirect (Elsevier - Open Access Articles via Crossref)
Document Type: article in journal/newspaper
Language: English
DOI: 10.1016/s1525-0016(16)33289-0
Availability: http://dx.doi.org/10.1016/s1525-0016(16)33289-0; https://api.elsevier.com/content/article/PII:S1525001616332890?httpAccept=text/plain; https://api.elsevier.com/content/article/PII:S1525001616332890?httpAccept=text/xml
Rights: https://www.elsevier.com/tdm/userlicense/1.0/ ; http://creativecommons.org/licenses/by-nc-nd/4.0/
Accession Number: edsbas.DDB8E119
Database: BASE