Published in Gene Therapy Weekly, March 31st, 2005
"Spinal muscular atrophy (SMA) is a frequent recessive autosomal disorder. It is caused by mutations or deletion of the telomeric copy of the survival motor neuron (SMN) gene, leading to depletion in SMN protein levels. The treatment rationale for SMA is to halt or delay the degeneration of motor neurons, but to date there are no effective drug treatments for this disease," scientists in England report.
"We have previously demonstrated that pseudotyping of the...
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