First-pass extracted concept

adeno-associated virus vector

Candidate: toolkit itemType: delivery harness2 source documents5 linked claims
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Aliases

AAV, AAV vector, AAV vectors, adeno-associated viruses

Extracted Explainers

What the tool is doing

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.

Source 1DOIPubMed

AAV vectors are presented as a major viral delivery system in gene therapy. The abstract links AAV to approved therapy and to dual-AAV strategies for large gene delivery.

Source 2DOIPubMed

Resources required

The reported use case requires intraperitoneal administration in mice and measurement of transgene expression over several weeks. The abstract does not specify the exact capsid, promoter, or payload.

Source 1DOIPubMed

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.

Source 1DOIPubMed

They provide a clinically relevant vector platform for gene delivery.

Source 2DOIPubMed

What it does not solve

The abstract states that AAV gene therapy is still limited by the cost and toxicity of the large doses required. It also does not establish whether the timing effect generalizes beyond the reported mouse intraperitoneal setting.

Source 1DOIPubMed

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.

Source 1DOIPubMed

The abstract contrasts AAV with lentiviral vectors, adenoviral vectors, LNP, and GalNAc-based non-viral approaches.

Source 2DOIPubMed

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.
Evidence 1Source 1DOIPubMedprovenance
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
Evidence 2Source 2DOIPubMedprovenance

Supporting Sources

Linked Claims

Claim 1clinical applicationsupports2025Source 2DOIPubMed

AAV-based gene therapy has achieved approved clinical use, including Luxturna for a genetic retinal disease.

Quoted textsource-backed
including the approval of Luxturna for a genetic retinal disease
Claim 2limitationsupports2025Source 1DOIPubMed

AAV vectors for gene therapy are limited by the cost and toxicity of the large doses required.

Quoted textsource-backed
The promise of adeno-associated virus (AAV) vectors for gene therapy is held back by the cost and toxicity of the large doses required.
Claim 3performance differencesupports2025Source 1DOIPubMed

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).

Quoted textsource-backed
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).
Claim 4translational implicationsupports2025Source 1DOIPubMed

Timing AAV administration by circadian phase could improve safety and reduce costs of AAV gene therapy and potentially other nanoparticle therapies.

Quoted textsource-backed
This insight could lead to future work that improves safety and reduces costs of AAV gene therapy and other nanoparticle therapies.
Claim 5utility overviewsupports2025Source 2DOIPubMed

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.

Quoted textsource-backed
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