The increasing number of approved nucleic acid therapeutics demonstrates the potential to
treat diseases by targeting their genetic blueprints in vivo. Conventional treatments …

Achieving sufficient delivery across the blood–brain barrier is a key challenge in the
development of drugs to treat central nervous system (CNS) disorders. This is particularly …

Despite scientific discoveries in the field of gene and cell therapy, some diseases still have
no effective treatment. Advances in genetic engineering methods have enabled the …

There has been a resurgence in gene therapy efforts that is partly fueled by the identification
and understanding of new gene delivery vectors. Adeno-associated virus (AAV) is a non …

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

Recombinant viruses are the workhorse of modern neuroscience. Whether one would like to
understand a neuron's morphology, natural activity patterns, molecular composition …

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 …

Both observational and perturbational technologies are essential for advancing the
understanding of brain function and dysfunction. But while observational techniques have …

Recombinant Adeno-associated virus vectors (rAAV) are widely used for gene delivery and
multiple naturally occurring serotypes have been harnessed to target cells in different …

Anxiety-related disorders can be treated by cognitive therapies and transcranial magnetic
stimulation, which involve the medial prefrontal cortex (mPFC). Subregions of the mPFC …