AAV vectors are used here as the gene therapy delivery platform whose in vivo transgene expression depends on dosing time. The paper studies how injection timing changes AAV efficacy in mice.
First-pass extracted concept
adeno-associated virus vector
Aliases
AAV, AAV vector, AAV vectors, adeno-associated viruses
Extracted Explainers
What the tool is doing
Resources required
What problem it solves
AAV provides a way to deliver gene therapy cargo and achieve sustained transgene expression in vivo. The study suggests that optimizing dosing time may improve this delivery platform's effective performance.
They provide a clinically relevant vector platform for gene delivery.
What it does not solve
Alternatives
The abstract mentions other nanoparticle therapies as a broader class that might also benefit from chronopharmacology. It does not name a specific alternative vector platform in the abstract itself.
The abstract contrasts AAV with lentiviral vectors, adenoviral vectors, LNP, and GalNAc-based non-viral approaches.
Evidence Snippets
The promise of adeno-associated virus (AAV) vectors for gene therapy is held back by the cost and toxicity of the large doses required.
Over the past two decades, three widely used viral vectors-lentiviruses (LV), adenoviruses (Ad), and adeno-associated viruses (AAV)-have enabled notable preclinical and clinical successes
Supporting Sources
Linked Claims
AAV-based gene therapy has achieved approved clinical use, including Luxturna for a genetic retinal disease.
including the approval of Luxturna for a genetic retinal disease
AAV vectors for gene therapy are limited by the cost and toxicity of the large doses required.
The promise of adeno-associated virus (AAV) vectors for gene therapy is held back by the cost and toxicity of the large doses required.
In mice, intraperitoneal AAV injection during the resting phase (ZT6) produced greater transgene expression over several weeks than equivalent injection during the waking phase (ZT18).
intraperitoneal doses of AAVs injected during the resting phase (ZT6, 12:00PM) produced greater transgene expression over several weeks than equivalent doses injected during the waking phase (ZT18, 12:00AM).
Timing AAV administration by circadian phase could improve safety and reduce costs of AAV gene therapy and potentially other nanoparticle therapies.
This insight could lead to future work that improves safety and reduces costs of AAV gene therapy and other nanoparticle therapies.
Lentiviral vectors, adenoviral vectors, and AAV are widely used viral vectors that have enabled notable preclinical and clinical successes in gene therapy over the past two decades.
Over the past two decades, three widely used viral vectors-lentiviruses (LV), adenoviruses (Ad), and adeno-associated viruses (AAV)-have enabled notable preclinical and clinical successes