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 …

Recent advancements in gene supplementation therapy are expanding the options for the
treatment of neurological disorders. Among the available delivery vehicles, adeno …

Background The goal of gene therapy for patients with hemophilia A is to safely impart long-
term stable factor VIII expression that predictably ameliorates bleeding with the use of the …

Massively parallel reporter gene assays are key tools in regulatory genomics but cannot be
used to identify cell-type-specific regulatory elements without performing assays serially …

Recombinant adeno-associated viruses (AAVs) have emerged as promising gene delivery
vehicles resulting in three US Food and Drug Administration (FDA) and one European …

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 …

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 …

Recombinant adeno-associated virus (rAAV) gene therapy has the potential to transform the
lives of patients with certain genetic disorders by increasing or restoring function to affected …

Host immune responses that limit durable therapeutic gene expression and cause clinically
significant inflammation remain a major barrier to broadly successful development of adeno …

In the past two decades, adeno-associated virus (AAV) vector manufacturing has made
remarkable advancements to meet large-scale production demands for preclinical and …