Duchenne muscular dystrophy (DMD) is the most common and most severe form of
muscular dystrophies and it is associated with progressive skeletal muscle degeneration …

Abstract Spinocerebellar ataxia type 1 (SCA1) is an autosomal dominant neurodegenerative
disease that causes progressive loss of motor coordination, respiratory issues, and eventual …

To date, more than 100 clinical trials have used sequence-based therapies to address
diseases of the pediatric central nervous system. The first targeted pathologies share …

Since the discovery of CRISPR-Cas9 systems, gene therapies have revolutionized the field
of molecular biology by introducing functional genes into cells to correct genetic defects or …

The clustered regularly interspaced short palindromic repeats (CRISPR)-Cas system, which
is an RNA-guided nuclease system, plays an important role in the adaptive immune …

In the January 13, 2023 issue of Science, Lebek and colleagues demonstrate the potential
broad utility of in vivo base editing as a gene therapy for heart disease [1]. Following …

Nature Communications describing immune responses to the Cas9 nuclease in several dog
models of Duchenne muscular dystrophy (DMD) undergoing gene editing therapy …

Duchenne muscular dystrophy (DMD) is an x-linked recessive lethal muscle wasting
disease with no cure. DMD is often caused by out-of-frame mutations that result in a loss of …

Merosin-deficient congenital muscular dystrophy (MDC1A) is an autosomal recessive
disorder caused by mutations in the LAMA2 gene, resulting in a defective form of the …