Datum der Indexierung 03.05.2024 22:09:36
erkannte Namen Gene Therapy |
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Kombination Keywords gene number | from muscular | this type | protein produced | deletion mrna | with have | clinical motor | exon patients | therapy disease | copies onset |
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Muscular Atrophy Background Fact Explanation Spinal muscular atrophy (SMA) Spinal muscular atrophy (SMA) refers to a group of rare genetic diseases, many of which are caused by deletions or mutations in both copies of the survival motor neuron 1 ( SM Adeno-associated virus In the gene therapy approach for the treatment of SMA, the exogenous SMN1 gene is stored in an adeno-associated virus (AAV9). This class of viruses is non-pathogenic to humans, causes a reduced immune responassociated virus In the gene therapy approach for the treatment of SMA, the exogenous SMN1 gene
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