Defective genes account for∼ 80% of the total of more than 7,000 diseases known to date.
Gene therapy brings the promise of a one-time treatment option that will fix the errors in …

CRISPR-based drugs can theoretically manipulate any genetic target. In practice, however,
these drugs must enter the desired cell without eliciting an unwanted immune response, so …

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 …

In the adult hippocampus, synapses are constantly formed and eliminated,. However, the
exact function of synapse elimination in the adult brain, and how it is regulated, are largely …

The success of base editors for the study and treatment of genetic diseases depends on the
ability to deliver them in vivo to the relevant cell types. Delivery via adeno-associated viruses …

Duchenne muscular dystrophy (DMD) is a fatal muscle disease caused by the lack of
dystrophin, which maintains muscle membrane integrity. We used an adenine base editor …

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

In recent years, the number of clinical trials in which adeno-associated virus (AAV) vectors
have been used for in vivo gene transfer has steadily increased. The excellent safety profile …

Adeno-associated viral (AAV) vectors are a rapidly emerging gene therapy platform for the
treatment of neurological diseases. In preclinical studies, transgenes encoding therapeutic …

We recently developed adeno-associated virus (AAV) capsids to facilitate efficient and
noninvasive gene transfer to the central and peripheral nervous systems. However, a …