Adeno-associated virus (AAV) vector-mediated gene delivery was recently approved for the
treatment of inherited blindness and spinal muscular atrophy, and long-term therapeutic …

The human genetic code encrypted in thousands of genes holds the secret for synthesis of
proteins that drive all biological processes necessary for normal life and death. Though the …

Genetically encoded voltage indicators (GEVIs) enable optical recording of electrical signals
in the brain, providing subthreshold sensitivity and temporal resolution not possible with …

Modern experimental technologies can assay large numbers of biological sequences, but
engineered protein libraries rarely exceed the sequence diversity of natural protein families …

Long-range gene editing by homology-directed repair (HDR) in hematopoietic
stem/progenitor cells (HSPCs) often relies on viral transduction with recombinant adeno …

Background Slowed clearance of amyloid β (Aβ) is believed to underlie the development of
Aβ plaques that characterize Alzheimer's disease (AD). Aβ is cleared in part by the …

Adeno-associated viruses (AAVs) are commonly used for in vivo gene transfer.
Nevertheless, AAVs that provide efficient transduction across specific organs or cell …

Optical interrogation of voltage in deep brain locations with cellular resolution would be
immensely useful for understanding how neuronal circuits process information. Here, we …

Recombinant adeno-associated virus (AAV) is the leading vector for gene therapy in the
retina. As non-pathogenic, non-integrating, replication deficient vector, the recombinant virus …

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