Publications by authors named "E K Gilbertson"
Inactivation of disease alleles by allele-specific editing is a promising approach to treat dominant-negative genetic disorders, provided the causative gene is haplo-sufficient. We previously edited a dominant missense mutation with inactivating frameshifts and rescued disease-relevant phenotypes in induced pluripotent stem cell (iPSC)-derived motor neurons. However, a multitude of different missense mutations cause disease.
View Article and Find Full Text PDFArticle Synopsis
- - Regulatory CD8 T cells (CD8 Treg) usually help control harmful CD4 T cells, but in autoimmune diseases, they often fail, partly due to inhibitory receptors that limit their activation.
- - A new bispecific antibody called MTX-101 targets these inhibitory receptors and CD8 T cells to enhance the ability of CD8 Treg to eliminate pathogenic CD4 T cells in various experimental models.
- - In studies, MTX-101 improved CD8 Treg activity, reduced harmful CD4 T cell expansion, and protected tissues from damage without triggering excessive inflammation, showing promise for treating autoimmune disorders.
Article Synopsis
- * A new machine learning method enables the quantification of 3D chromatin contacts from genome sequences, revealing notable differences in contact and sequence divergence, particularly in specific genomic windows.
- * The research shows that in regions with high 3D divergence, certain genetic variants can lead to significant changes in chromatin contact without corresponding changes in the DNA sequence, highlighting the complexity of genetic variation impacts.
The 3D structure of the genome is an important mediator of gene expression. As phenotypic divergence is largely driven by gene regulatory variation, comparing genome 3D contacts across species can further understanding of the molecular basis of species differences. However, while experimental data on genome 3D contacts in humans are increasingly abundant, only a handful of 3D genome contact maps exist for other species.
View Article and Find Full Text PDF