Frame-disrupting mutations in the DMD gene, encoding dystrophin, compromise myofiber
integrity and drive muscle deterioration in Duchenne muscular dystrophy (DMD). Removing …

CRISPR–Cas9 delivery by adeno-associated virus (AAV) holds promise for gene therapy
but faces critical barriers on account of its potential immunogenicity and limited payload …

Many genetic diseases are caused by single-nucleotide polymorphisms. Base editors can
correct these mutations at single-nucleotide resolution, but until recently, only allowed for …

Mito-SEPs are small open reading frame-encoded peptides that localize to the mitochondria
to regulate metabolism. Motivated by an intriguing negative association between mito-SEPs …

Genome‐editing therapeutics are poised to treat human diseases. As we enter clinical trials
with the most promising CRISPR‐Cas9 and CRISPR‐Cas12a (Cpf1) modalities, the risks …

Background Human respiratory epithelia function in airway mucociliary clearance and barrier
function and have recently been implicated in sensory functions. Objective We investigated …

Mutations in the LaminA gene are a common cause of monogenic dilated cardiomyopathy.
Here we show that mice with a cardiomyocyte-specific Lmna deletion develop cardiac failure …

Protein-based therapeutics can activate the adaptive immune system, leading to the production
of neutralizing antibodies and the clearance of the treated cells mediated by cytotoxic T …

Precise editing is essential for biomedical research and gene therapy. Yet, homology-directed
genome modification is limited by the requirements for genomic lesions, homology donors …

Background: Epicardial adipose tissue volume and coronary artery disease are strongly
associated, even after accounting for overall body mass. Despite its pathophysiological …