CAG-FLEX-NES-jRCaMP1b AAV (Serotype 8)

Genetic diseases account for more than 70% of hospital admissions in children and more than 10% of hospital admissions in adults. These genetic diseases often require lifelong treatment and are a significant expense to the healthcare system and society as a whole. One of the most promising and well-researched long-term treatment strategies for these diseases is gene therapy, which involves delivering functional genes into cells to supplement or replace missing or defective genes to correct/ameliorate the disease. Adeno-associated virus (AAV) is emerging as one of the preferred vectors for therapeutic gene transfer. AAV belongs to the Parvoviridae family and consists of a single-stranded DNA genome of approximately 4.7 kb encapsidated in an icosahedral viral capsid.

AAVs have emerged as potential delivery vectors because they have five specific features that facilitate safe and effective gene delivery: (1) certain AAV strains are not associated with serious disease; (2) AAVs can be engineered to be essentially devoid of viral DNA; (3) they can stably express many genes in vivo while inducing limited immune responses to the vector or transgene; (4) they have broad and promiscuous tropisms; and (5) they can achieve efficient and long-lasting gene transfer. Together, these features illustrate the capabilities of AAVs as gene therapy vectors.