Duchenne muscular dystrophy (DMD) is a monogenic disorder and a candidate for
therapeutic genome editing. There have been several recent reports of genome editing in …

Targeted genome editing via engineered nucleases is an exciting area of biomedical
research and holds potential for clinical applications. Despite rapid advances in the field, in …

There is a significant interest in identifying blood-borne factors that mediate tissue crosstalk
and function as molecular effectors of physical activity. Although past studies have focused …

Current genome-editing systems generally rely on inducing DNA double-strand breaks
(DSBs). This may limit their utility in clinical therapies, as unwanted mutations caused by …

Dravet syndrome is a severe epileptic encephalopathy caused primarily by
haploinsufficiency of the SCN1A gene. Repetitive seizures can lead to endurable and …

During development, melanopsin-expressing intrinsically photosensitive retinal ganglion
cells (ipRGCs) become light sensitive much earlier than rods and cones. IpRGCs project to …

Challenges to the widespread application of gene therapy with adeno-associated viral
(AAV) vectors include dominant conditions due to gain-of-function mutations which require …

Although the classic symptoms of Huntington's disease (HD) manifest in adulthood, neural
progenitor cell behavior is already abnormal by 13 weeks' gestation. To determine how …

Transcription of expanded microsatellite repeats is associated with multiple human
diseases, including myotonic dystrophy, Fuchs endothelial corneal dystrophy, and C9orf72 …

Targeted gene-editing strategies have emerged as promising therapeutic approaches for
the permanent treatment of inherited genetic diseases. However, precise gene correction …