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onasemnogene abeparvovec-xio (Zolgensma)
Indications:
- gene therapy medication used to treat spinal muscular atrophy
Dosage:
- administered intravenously or intrathecally Mechanism:
- biologic drug consisting of AAV9 virus capsids deprived original viral DNA, instead contain a SMN1 transgene along with promoters
- the SMN1 transgene is delivered to neuronal cell nuclei by the AAV9 viral vector
- transcription of SMN1 gene begins with translation of resultant mRNA to produce a functional SMN protein
- the procedure addresses the root cause of spinal muscular atrophy, lack of a functional SMN protein
- motor neurons do not divide, thus a single dose of Zolgensma is expected to have a lifelong effect
General
neurologic agent
References
- Wikipedia: Onasemnogene abeparvovec
https://en.wikipedia.org/wiki/Onasemnogene_abeparvovec