The development of new CRISPR–Cas genome editing tools continues to drive major
advances in the life sciences. Four classes of CRISPR–Cas-derived genome editing agents …

Gene editing that makes target gene modification in the genome by deletion or addition has
revolutionized the era of biomedicine. Clustered regularly interspaced short palindromic …

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

Spinal muscular atrophy (SMA), the leading genetic cause of infant mortality, arises from
survival motor neuron (SMN) protein insufficiency resulting from SMN1 loss. Approved …

Genome editing therapy for Duchenne muscular dystrophy (DMD) holds great promise,
however, one major obstacle is delivery of the CRISPR-Cas9/sgRNA system to skeletal …

The prokaryote-derived CRISPR–Cas genome editing systems have transformed our ability
to manipulate, detect, image and annotate specific DNA and RNA sequences in living cells …

To the Editor—The field of genome editing is advancing rapidly1, most recently exemplified
by the advent of base editors that enable changing single nucleotides in a predictable …

Recombinant adeno-associated viruses (AAVs) are commonly used gene delivery vehicles
for neuroscience research. They have two engineerable features: the capsid (outer protein …

Mutations in the gene encoding dystrophin, a protein that maintains muscle integrity and
function, cause Duchenne muscular dystrophy (DMD). The deltaE50-MD dog model of DMD …

Within less than a decade since its inception, CRISPR-Cas9-based genome editing has
been rapidly advanced to human clinical trials in multiple disease areas. Although it is highly …