Astrocyte targeted SMN1 gene therapy and forskolin application improves astrocyte filopodia actin defects and motor neuron synaptic dysfunction in human SMA disease pathology
Emily Welby, Xiaojie Liu, Melinda S. Wojtkiewicz…·Allison D. Ebert
Author
Medical College of WisconsinORCID 0000-0003-2408-0407Unclaimed ORCID profile · follow to get notified of new preprints
6 papers
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Emily Welby, Xiaojie Liu, Melinda S. Wojtkiewicz…·Allison D. Ebert
This study identifies a non-cell autonomous mechanism in SMA where astrocytic GLAST dysfunction contributes to motor neuron dysfunction, which is partially restored by SMN re-expression.
Reilly L. Allison, Emily Welby, Guzal Khayrullina…·Allison D. Ebert
Emily Welby, Rebecca Rehborg, Matthew M. Harmelink, Allison D. Ebert
This study assesses CSF biomarkers and microRNA-mediated mechanisms in SMA patients treated with nusinersen, finding improved microRNA expression but persistent cellular stress and astrocyte dysfunction.
Samuel W. Lukowski, Camden Yeung-Wah Lo, Alexei A. Sharov…·Raymond C.B. Wong
The paper generates a human neural retina transcriptome atlas using single-cell RNA sequencing, identifying 18 cell types and revealing a novel role for MALAT1 in rod degeneration.
Koray Dogan Kaya, Holly Y. Chen, Matthew J. Brooks…·Anand Swaroop
Comparing retinal organoid transcriptomes against human fetal and adult retina places each culture on a developmental scale; replacing all-trans retinoic acid with 9-cis retinal sped rod differentiation.