Aging is driven by hallmarks fulfilling the following three premises:(1) their age-associated
manifestation,(2) the acceleration of aging by experimentally accentuating them, and (3) the …

The advent of clustered regularly interspaced short palindromic repeat (CRISPR) genome
editing, coupled with advances in computing and imaging capabilities, has initiated a new …

In vivo gene editing therapies offer the potential to treat the root causes of many genetic
diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to …

The targeted deletion, replacement, integration or inversion of genomic sequences could be
used to study or treat human genetic diseases, but existing methods typically require double …

Methods to deliver gene editing agents in vivo as ribonucleoproteins could offer safety
advantages over nucleic acid delivery approaches. We report the development and …

Dominant missense pathogenic variants in cardiac myosin heavy chain cause hypertrophic
cardiomyopathy (HCM), a currently incurable disorder that increases risk for stroke, heart …

Gene-editing technologies, which include the CRISPR–Cas nucleases 1, 2, 3 and CRISPR
base editors 4, 5, have the potential to permanently modify disease-causing genes in …

The most common form of genetic heart disease is hypertrophic cardiomyopathy (HCM),
which is caused by variants in cardiac sarcomeric genes and leads to abnormal heart …

Realizing the promise of prime editing for the study and treatment of genetic disorders
requires efficient methods for delivering prime editors (PEs) in vivo. Here we describe the …

The viral delivery of base editors has been complicated by their size and by the limited
packaging capacity of adeno-associated viruses (AAVs). Typically, dual-AAV approaches …