Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the
treatment of a variety of human diseases. Recent advances in developing clinically …

Amyotrophic lateral sclerosis (ALS) has historically posed unique challenges for gene-
therapy-based approaches, due to a paucity of therapeutic targets as well as the difficulty of …

Neurotropic adeno-associated virus (AAV) serotypes such as AAV9 have been
demonstrated to transduce spinal alpha motor neurons when administered intravenously (iv) …

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

Two patients with familial amyotrophic lateral sclerosis (ALS) and mutations in the gene
encoding superoxide dismutase 1 (SOD1) were treated with a single intrathecal infusion of …

Cell-type-specific expression of molecular tools and sensors is critical to construct circuit
diagrams and to investigate the activity and function of neurons within the nervous system …

Cas13 nucleases are a class of programmable RNA-targeting CRISPR effector proteins that
are capable of silencing target gene expression in mammalian cells. Here, we demonstrate …

Gene therapy is at the cusp of a revolution for treating a large spectrum of CNS disorders by
providing a durable therapeutic protein via a single administration. Adeno-associated virus …

Amyotrophic lateral sclerosis (ALS) is the most common adult-onset motor neuron disease
and is characterized by the degeneration of upper and lower motor neurons. It has become …

Amyotrophic lateral sclerosis (ALS) is a fatal, multigenic, multifactorial, and non-cell
autonomous neurodegenerative disease characterized by upper and lower motor neuron …