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March 14, 2017Molecular Therapy — Nucleic AcidsOpen Access

Efficient SMN Rescue following Subcutaneous Tricyclo-DNA Antisense Oligonucleotide Treatment

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Why the study?

Does subcutaneous tricyclo-DNA antisense oligonucleotide treatment improve SMN2 mRNA splicing and disease phenotype in mild type III SMA mice?

Population

Mild type III SMA mice

Design

Preclinical

Authors

VRValérie RobinUniversité de Versailles Saint-Quentin-en-YvelinesGGGraziella GriffithInsermJCJohn-Paul L. Carter

Discussion

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Implication

Suggests systemic antisense oligonucleotide delivery can cross the blood-brain barrier in animal models; leaves open whether.

Structured PICO

Does subcutaneous tricyclo-DNA antisense oligonucleotide treatment improve SMN2 mRNA splicing and disease phenotype in mild type III SMA mice?

P
Population
Mild type III SMA mice (selected to test efficacy and ability to enter the CNS after maturation of the blood brain barrier)
I
Intervention
Subcutaneous systemic delivery of tricyclo-DNA (tcDNA) antisense oligonucleotides (AONs)
O
Outcome
Retention of exon 7 in SMN2 mRNA in peripheral organs and the CNS, necrosis phenotype, and respiratory functionsurrogate

Systemic subcutaneous delivery of tcDNA AONs effectively crosses the blood-brain barrier and improves disease phenotype in a mouse model of mild type III SMA, offering a potential systemic therapeutic alternative.

Cite This Study

Robin et al. (2017) studied this question.

synapsesocial.com/papers/6a836e71af85195529c94b4ehttps://doi.org/10.1016/j.omtn.2017.02.009
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