Therapeutic approaches for spinal muscular atrophy (SMA).

Scoto, M; Finkel, R S; Mercuri, E; Muntoni, F

Scoto, M; Finkel, R S; Mercuri, E; Muntoni, F.

Affiliation

Scoto M; The Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK.
Finkel RS; Department of Pediatric Neurology, Nemours Children's Hospital, College of Medicine, University of Central Florida, Orlando, FL, USA.
Mercuri E; Department of Pediatric Neurology, Catholic University and Centro Nemo, Policlinico Gemelli, Rome, Italy.
Muntoni F; The Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK.

Gene Ther ; 24(9): 514-519, 2017 09.

Article in En | MEDLINE | ID: mdl-28561813

Subject(s)

Genetic Therapy/methods; Muscular Atrophy, Spinal/therapy; Animals; Clinical Trials as Topic; Genetic Therapy/adverse effects; Humans; Muscular Atrophy, Spinal/genetics; Oligonucleotides, Antisense/administration & dosage; Survival of Motor Neuron 1 Protein/genetics; Survival of Motor Neuron 1 Protein/metabolism; Survival of Motor Neuron 2 Protein/genetics; Survival of Motor Neuron 2 Protein/metabolism

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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Muscular Atrophy, Spinal / Genetic Therapy Type of study: Prognostic_studies Limits: Animals / Humans Language: En Journal: Gene Ther Journal subject: GENETICA MEDICA / TERAPEUTICA Year: 2017 Type: Article Affiliation country: United kingdom

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