Current developments in gene therapy for amyotrophic lateral sclerosis.

Scarrott, Joseph M; Herranz-Martín, Saúl; Alrafiah, Aziza R; Shaw, Pamela J; Azzouz, Mimoun

Scarrott, Joseph M; Herranz-Martín, Saúl; Alrafiah, Aziza R; Shaw, Pamela J; Azzouz, Mimoun.

Affiliation

Scarrott JM; University of Sheffield, Sheffield Institute for Translational Neuroscience (SITraN) , 385 Glossop Road, Sheffield, S10 2HQ , UK.

Expert Opin Biol Ther ; 15(7): 935-47, 2015 Jul.

Article in En | MEDLINE | ID: mdl-25959569

Subject(s)

Amyotrophic Lateral Sclerosis/therapy; Genetic Therapy; Amyotrophic Lateral Sclerosis/genetics; Amyotrophic Lateral Sclerosis/physiopathology; Animals; C9orf72 Protein; Dependovirus/genetics; Disease Models, Animal; Humans; Motor Neurons/metabolism; Motor Neurons/pathology; Nerve Degeneration; Nerve Growth Factors/genetics; Nerve Growth Factors/metabolism; Proteins/genetics; Proteins/metabolism; RNA Interference; Superoxide Dismutase/genetics; Superoxide Dismutase/metabolism; Superoxide Dismutase-1

Key words

adeno-associated virus; amyotrophic lateral sclerosis; gene therapy; self-complementary adeno-associated virus serotype 9

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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Genetic Therapy / Amyotrophic Lateral Sclerosis Type of study: Prognostic_studies Limits: Animals / Humans Language: En Journal: Expert Opin Biol Ther Journal subject: BIOLOGIA / TERAPEUTICA Year: 2015 Document type: Article Affiliation country: United kingdom

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