Also: adeno-associated virus, viral vectors, gene therapy
Adeno-associated virus (AAV) is a harmless virus that scientists empty and use as a delivery vehicle for genes. It underpins several approved gene therapies.
Readiness: lab lab studies · prototype demonstrated objects · commercial products or buildings in real use · review summaries of other studies.
Maguire and colleagues delivered a working RPE65 gene by AAV to three patients with Leber congenital amaurosis; it appeared safe and gave modest improvements in retinal function.
As reported in: Maguire, Simonelli, Pierce et al. (2008). Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis. New England Journal of Medicine. doi:10.1056/NEJMoa0802315
Russell and colleagues reported the phase 3 trial of an AAV gene therapy for RPE65 blindness, later approved as Luxturna.
Built / tested: Luxturna
As reported in: Russell, Bennett, Wellman et al. (2017). Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. The Lancet. doi:10.1016/S0140-6736(17)31868-8
Mendell and colleagues gave infants with SMA a single AAV gene-replacement dose, greatly improving survival.
Built / tested: onasemnogene (Zolgensma)
As reported in: Mendell, Al-Zaidy, Shell et al. (2017). Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy. New England Journal of Medicine. doi:10.1056/NEJMoa1706198
Wang, Tai and Gao reviewed AAV as a platform for gene therapy delivery.
As reported in: Wang, Tai, Gao (2019). Adeno-associated virus vector as a platform for gene therapy delivery. Nature Reviews Drug Discovery. doi:10.1038/s41573-019-0012-9