DG9-conjugated morpholino rescues phenotype in SMA mice by reaching the CNS via a subcutaneous administration-Reference-Cited by-同舟云学术

DG9-conjugated morpholino rescues phenotype in SMA mice by reaching the CNS via a subcutaneous administration

Published:2023-03-08 Issue:5 Volume:8 Page:
ISSN:2379-3708
Container-title:JCI Insight
language:en
Short-container-title:

Author:

Aslesh Tejal^ORCID,Erkut Esra^ORCID,Ren Jun,Lim Kenji Rowel Q.^ORCID,Woo Stanley^ORCID,Hatlevig Susan,Moulton Hong M.,Gosgnach Simon,Greer John,Maruyama Rika^ORCID,Yokota Toshifumi

Funder

Canadian Institutes of Health Research

Women and Children’s Health Research Institute

The Friends of Garrett Cumming Research & Muscular Dystrophy Canada HM Toupin Neurological Science Research Chair

SMA Slipchuk Foundation

Publisher

American Society for Clinical Investigation

Subject

General Medicine

Link

https://insight.jci.org/articles/view/160516/files/pdf

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5. Inactivation of the survival motor neuron gene, a candidate gene for human spinal muscular atrophy, leads to massive cell death in early mouse embryos

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