Precise genetic modifications in model animals are essential for biomedical research. Here,
we report a programmable “base editing” system to induce precise base conversion with …

Introduction The inability of some seriously and chronically ill individuals to receive a
definitive diagnosis represents an unmet medical need. In 2008, the NIH Undiagnosed …

Defects in patterning during human embryonic development frequently result in craniofacial
abnormalities. The gene regulatory programs that build the craniofacial complex are likely …

Background: Microtia is a congenital malformation of the outer ears caused by improper
embryonic development. The origin of microtia and causes of its variations remain unknown …

Background To review and update the syndromic and non-syndromic systemic associations
of congenital lacrimal drainage anomalies. Methods The authors performed a PubMed …

Background Gene targeting by homology-directed repair (HDR) can precisely edit the
genome and is a versatile tool for biomedical research. However, the efficiency of HDR …

Gorlin-Chaudhry-Moss syndrome (GCMS) is a dysmorphic syndrome characterized by
coronal craniosynostosis and severe midface hypoplasia, body and facial hypertrichosis …

Network medicine approaches have been largely successful at increasing our knowledge of
molecularly characterized diseases. Given a set of disease genes associated with a …

Eukaryotic transcription factors recognize specific DNA sequence motifs, but are also
endowed with generic, non‐specific DNA‐binding activity. How these binding modes are …

Dear Editor, Point mutations are the cause of many human genetic diseases 1. However, the
typical indel mutations generated by CRISPR/Cas9 make this system not ideal for modeling …