Daifeng Wang, PhD – Slide of the Week

Understanding cell-type-specific gene regulatory mechanisms from genetic variants to diseases remains challenging. To address this, we developed a computational pipeline, scGRNom (single-cell Gene Regulatory Network prediction from multi-omics), to predict cell-type disease genes and regulatory networks including transcription factors and regulatory elements.

John Svaren, PhD – Slide of the Week

Title: CRISPR-Cas9 deletion of PMP22 super enhancer creates  a new model of hereditary neuropathy with liability to pressure palsies (HNPP) Legend: (A) Morphometric analysis demonstrates axonal loss i thin section were taken from 3- to 5-month-old …

Audra Sterling, PhD – Slide of the Week

Autism spectrum disorder (ASD) and fragile X syndrome (FXS) are neurodevelopmental disorders with overlapping pragmatic language impairments. Prior work suggests pragmatic language differences may run in families. This study examined specific pragmatic difficulties (i.e., linguistic mazes and perseverations) in boys (9–18 years) with idiopathic ASD (n = 26) and FXS+ASD (n = 29), and relationships with maternal maze use.

Marsha R. Mailick, PhD – Slide of the Week

 Fragile X syndrome (FXS), the most prevalent inherited cause of intellectual disability, remains under-diagnosed in the general population. Clinical studies have shown that individuals with FXS have a complex health profile leading to unique clinical needs. However, the full impact of this X-linked disorder on the health of affected individuals is unclear and the prevalence of co-occurring conditions is unknown.

Krishanu Saha, PhD – Slide of the Week

The move from reading to writing the human genome offers new opportunities to improve human health. The United States National Institutes of Health (NIH) Somatic Cell Genome Editing (SCGE) Consortium aims to accelerate the development of safer and more effective methods to edit the genomes of disease-relevant somatic cells in patients, even in tissues that are difficult to reach.

Bernadette Gillick, PhD, MSPT, PT – Slide of the Week

Cerebral palsy is caused by a congenital brain lesion that occurs early in life with associated motor deficits which may result in lifelong disability. The brain has high neuroplastic potential early in life, stressing the importance for therapy. Non-invasive brain stimulation (NIBS) including transcranial direct current stimulation (tDCS) may enhance pediatric rehabilitation interventions through neuroplasticity.