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 …

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 …

Recent advances in genome editing technologies have magnified the prospect of single-
dose cures for many genetic diseases. For most genetic disorders, precise DNA correction is …

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

Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene
therapy owing to its minimal pathogenicity and ability to establish long-term gene expression …

The engineered AAV-PHP. B family of adeno-associated virus efficiently delivers genes
throughout the mouse central nervous system. To guide their application across disease …

Adeno-associated virus (AAV) is the leading vector in emerging treatments of inherited
diseases. Higher transduction efficiencies and cellular specificity are required for broader …

Viral tracers are important tools for neuroanatomical mapping and genetic payload delivery.
Genetically modified viruses allow for cell-type-specific targeting and overcome many …

Adeno-associated virus (AAV) is a highly promising gene transfer vector, yet major cellular
requirements for AAV entry are poorly understood. Using a genome-wide CRISPR screen …

Gene therapy has garnered increasing interest over recent decades. Several therapies
employing gene transfer mechanisms have been developed, and, of these, adeno …