Microglia play essential yet poorly understood roles in brain development, including axon guidance, regulation of neurogenesis, and pruning of neuronal projections. Congenital hydrocephalus (CH), characterized by enlarged cerebrospinal fluid (CSF)-filled ventricles, is a leading cause of pediatric brain surgery, but it...
Phan Q. Duy, Benjamin C. Reeves, Huan-Xing Sun et al.· Journal of Clinical Investig...· 0 citations
This Perspective by the LRS-RED consortium discusses methodological, bioinformatic and diagnostic advances in long-read sequencing (LRS) for repeat-expansion disorders, highlighting the potential of LRS to reshape research and clinical practice.
L. Benarroch, J. Pešović, Marzia Rossato et al.· Nature Genetics· 0 citations
A reliable prognostic distinction between benign and pathogenic CTDs is defined and a potential editing strategy for correcting disease-causing CTD mutations is established.
J. Guy, E. Hein, Beatrice K. Alexander-Howden et al.· eLife· 0 citations
It is concluded from preliminary data that gene therapy using BE HSPCs appear very promising for achieving an earlier and more robust multi-lineage immune reconstitution in adult patients using BE HSPCs.
S. S. de Ravin, Michelle Ma, Yuzhi Yin et al.· Journal of Human Immunity· 0 citations
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