Progress in strategies aimed at breaking down therapeutic target proteins has led to a
paradigm shift in drug discovery. Thalidomide and its derivatives are the only protein …

Congenital heart disease is the leading cause of infant morbidity in the Western world, but
only in the past ten years has its aetiology been understood. Recent studies have uncovered …

Targeted protein degradation via small-molecule modulation of cereblon offers vast potential
for the development of new therapeutics. Cereblon-binding therapeutics carry the safety …

The mechanisms controlling axon guidance are of fundamental importance in
understanding brain development. Growing corticospinal and somatosensory axons cross …

Objective: To investigate the variable clinical picture of Möbius syndrome (MIM no.
157900) and to further understand the pathogenesis of the disorder. Methods: A …

Advances in neuroimaging, developmental biology and molecular genetics have increased
the understanding of developmental disorders affecting the midbrain and hindbrain, both as …

Thalidomide, originally developed as a sedative drug, causes multiple defects due to severe
teratogenicity, but it has been re-purposed for treating multiple myeloma, and derivatives …

Congenital heart disease (CHD) affects nearly 1% of the population. It is a complex disease,
which may be caused by multiple genetic and environmental factors. Studies in human …

Mutations in SALL4, the human homolog of the Drosophila homeotic gene spalt (sal), cause
the autosomal dominant disorder known as Okihiro syndrome. In this study, we show that a …

SALL4, a human homolog to Drosophila spalt, is a novel zinc finger transcriptional factor
essential for development. We cloned SALL4 and its isoforms (SALL4A and SALL4B) …