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Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

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

in Age / Children / Children & youth / Cohort Studies / Complementary DNA / Deoxyribonucleic acid / Dependovirus / Disease-Free Survival / DNA / Female / Gene therapy / Gene transfer / Genetic Therapy - adverse effects / Genetic Vectors / Historically Controlled Study / Humans / Infant / Infant, Newborn / Infusions, Intravenous / Intravenous administration / Liver Diseases - etiology / Male / Motor neuron diseases / Motor Skills / Nervous system / Neuromuscular diseases / Neurons / Nutritional Support / Patients / Prednisolone / Proteins / Respiration, Artificial / SMN protein / Spinal Muscular Atrophies of Childhood - genetics / Spinal Muscular Atrophies of Childhood - physiopathology / Spinal Muscular Atrophies of Childhood - therapy / Spinal muscular atrophy / Survival of Motor Neuron 1 Protein - genetics

2017

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2017

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2017

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Journal Article

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

Braun, Lyndsey,

Foust, K.D,

Meyer, Kathrin,

Al-Zaidy, Samiah,

Alfano, Lindsay,

Wells, Courtney,

Nagendran, Sukumar,

Kaspar, Allan,

Rodino-Klapac, Louise R,

Berry, Katherine,

Arnold, W. Dave,

Corcoran, Sarah,

Prior, Thomas W,

Kaspar, Brian K,

L’Italien, James,

Sproule, Douglas M,

Mendell, Jerry R,

Likhite, Shibi,

Church, Kathleen,

Lowes, Linda,

Cardenas, Jessica A,

Miranda, Carlos,

Kissel, John T,

Burghes, Arthur H.M,

Heitzer, Marjet D,

Shell, Richard

2017

Overview

Fifteen children with spinal muscular atrophy type 1 received gene-replacement therapy with a single dose of adeno-associated virus containing SMN. In marked contrast to well-characterized historical cohorts, all the patients survived at least 20 months and most reached motor milestones.

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Publisher

Massachusetts Medical Society

Subject

/ Deoxyribonucleic acid

/ Dependovirus