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Comment
. 2022 May 17:16:916065.
doi: 10.3389/fncel.2022.916065. eCollection 2022.

Commentary: Current Status of Gene Therapy for Spinal Muscular Atrophy

Wilfried Rossoll  1 , Ravindra N Singh  2
Affiliations
Comment

Commentary: Current Status of Gene Therapy for Spinal Muscular Atrophy

Wilfried Rossoll et al. Front Cell Neurosci. .
No abstract available

Keywords: AAV9; AVXS-101; Survival Motor Neuron (SMN); Zolgensma; gene therapy; onasemnogene abeparvovec; spinal muscular atrophy (SMA).

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Conflict of interest statement

The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

Figures

Figure 1
Figure 1
Strategies for increasing therapeutic transduction efficiency and reducing toxicity. Concerns raised by recent studies and future strategies to address these challenges as they relate to the route of administration, the AAV serotype/capsid, and expression construct are summarized. IV, intravenous; ICV, intracerebroventricular; ICM, intracisterna magna, IT, intrathecal; CMV, cytomegalovirus; miRNA, micro-RNA. Figure created with BioRender.com.
See this image and copyright information in PMC

Comment on

References

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