Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy | Zendy

Jerry R. Mendell | Zendy; Samiah Al-Zaidy | Zendy; Richard Shell | Zendy; W. David Arnold | Zendy; Louise R. RodinoKlapac | Zendy; Thomas W. Prior | Zendy; Linda Lowes | Zendy; Lindsay N. Alfano | Zendy; Katherine Berry | Zendy; Kathleen Church | Zendy; John T. Kissel | Zendy; Sukumar Nagendran | Zendy; James L’Italien | Zendy; Douglas M. Sproule | Zendy; Courtney Wells | Zendy; Jessica Cardenas | Zendy; Marjet D. Heitzer | Zendy; Allan Kaspar | Zendy; Sarah Corcoran | Zendy; Lyndsey Braun | Zendy; Shibi Likhite | Zendy; Carlos J. Miranda | Zendy; Kathrin Meyer | Zendy; Kevin D. Foust | Zendy; Arthur H.M. Burghes | Zendy; Brian K. Kaspar | Zendy

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Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

Author(s) -

Jerry R. Mendell,

Samiah Al-Zaidy,

Richard Shell,

W. David Arnold,

Louise R. RodinoKlapac,

Thomas W. Prior,

Linda Lowes,

Lindsay N. Alfano,

Katherine Berry,

Kathleen Church,

John T. Kissel,

Sukumar Nagendran,

James L’Italien,

Douglas M. Sproule,

Courtney Wells,

Jessica Cardenas,

Marjet D. Heitzer,

Allan Kaspar,

Sarah Corcoran,

Lyndsey Braun,

Shibi Likhite,

Carlos J. Miranda,

Kathrin Meyer,

Kevin D. Foust,

Arthur H.M. Burghes,

Brian K. Kaspar

Publication year - 2017

Publication title -

new england journal of medicine

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 19.889

H-Index - 1030

eISSN - 1533-4406

pISSN - 0028-4793

DOI - 10.1056/nejmoa1706198

Subject(s) - smn1 , spinal muscular atrophy , medicine , motor neuron , progressive muscular atrophy , atrophy , disease , neuromuscular disease , genetic enhancement , sma* , physical medicine and rehabilitation , bioinformatics , amyotrophic lateral sclerosis , pathology , gene , genetics , mathematics , combinatorics , biology

Spinal muscular atrophy type 1 (SMA1) is a progressive, monogenic motor neuron disease with an onset during infancy that results in failure to achieve motor milestones and in death or the need for mechanical ventilation by 2 years of age. We studied functional replacement of the mutated gene encoding survival motor neuron 1 (SMN1) in this disease.

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