https://openalex.org/T12400
This cluster of papers focuses on the gene therapy, molecular genetics, and clinical management of Spinal Muscular Atrophy (SMA). It covers topics such as SMN1 and SMN2 genes, Nusinersen treatment, antisense oligonucleotide therapies, motor neuron pathology, and the role of survival motor neuron protein in SMA. The cluster also discusses potential therapeutic strategies, including gene replacement therapy and the use of small molecules to modulate SMN expression.
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skos:definition "This cluster of papers focuses on the gene therapy, molecular genetics, and clinical management of Spinal Muscular Atrophy (SMA). It covers topics such as SMN1 and SMN2 genes, Nusinersen treatment, antisense oligonucleotide therapies, motor neuron pathology, and the role of survival motor neuron protein in SMA. The cluster also discusses potential therapeutic strategies, including gene replacement therapy and the use of small molecules to modulate SMN expression."@en ;
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openalex:cited_by_count 414105 ;
openalex:works_count 33899 .