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AAV based gene therapy for rare diseases

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AAV based gene therapy for rare diseases

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Efficient transduction of non-human primate motor neurons after intramuscular delivery of recombinant AAV serotype 6

Neuroprotection by Gene Therapy Targeting Mutant SOD1 in Individual Pools of Motor Neurons Does not Translate Into Therapeutic Benefit in fALS Mice

AAV-mediated expression of wild-type and ALS-linked mutant VAPB selectively triggers death of motoneurons through a Ca2+-dependent ER-associated pathway

Collapsin response médiator protein 4a(CRMP 4a) is upregulated in motoneurons of mutant SOD1 mice and can trigger motoneuron death

Lentiviral and Adeno-Associated Vector-Based Therapy for Motor Neuron Disease Through RNAi

Diagnosis of muscular dystrophies at the nanometer scale

Isolation and evaluation of novel adeno-associated virus sequences from porcine tissues

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

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

Conditional gene expression and knockdown using lentivirus vectors encoding shRNA