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Transgenic SOD1 mice as a model for developing amyotrophic lateral sclerosis therapies

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Transgenic SOD1 mice as a model for developing amyotrophic lateral sclerosis therapies

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On the replication of adeno-associated virus in the presence or absence of viral helper functions

Response of aged parkinsonian monkeys to in vivo gene transfer of GDNF

Conditional gene expression and knockdown using lentivirus vectors encoding shRNA

Systemic AAV6 delivery mediating RNA interference against SOD1: neuromuscular transduction does not alter disease progression in fALS mice

Recombinant Adeno-Associated viral vectors are deficient in provoking a DNA damage response

Dependence of reversibility and progression of mouse neuronopathic Gaucher disease on acid beta-glucosidase residual activity levels

GDNF secreting human neural progenitor cells protect dying motor neurons, but not their projection to muscle, in a rat model of familial ALS

Lentiviral vector-mediated genetic modification of human neural progenitor cells for ex vivo gene therapy

Metabolic correction in oligodendrocytes derived from metachromatic leukodystrophy mouse model by using encapsulated recombinant myoblasts

Inhibition of HIV-1 multiplication by a modified U7 snRNA inducing Tat and Rev exon skipping