ArticleMolecular therapy. Methods & clinical development2024
A novel class of self-complementary AAV vectors with multiple advantages based on cceAAV lacking mutant ITR.
Article in Molecular therapy. Methods & clinical development, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.
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Who cites it
12 citing papers in PubMed, 13 citations in OpenAlex.
- Orthogonal characterization of rAAV reveals vector attributes that drive ITR repair, self-complementary genome formation, and transgene expression.Molecular therapy. Nucleic acids · 2026Article
- Safety and Efficacy of cceAAV-aflibercept-Fc-YTE (F-CRG-B191): A Gene Therapy for Neovascular Age-Related Macular Degeneration.Investigative ophthalmology & visual science · 2026Article
- Rational engineering of the P5 TRS-mimic site and REP78/68 start codon yields promoter variants that improve rAAV purity while maintaining high titers.Molecular therapy. Advances · 2026Article
- Genetic medicines for epilepsy: unlocking new avenues for seizure control.Frontiers in bioengineering and biotechnology · 2026Review
- Advances in endothelial cell targeting by AAV vectors.Acta biochimica Polonica · 2026Review
- Chronologically distributed transfection improves AAV2 and AAV2/8 capsid filling and reveals assembly schedule divergence.Molecular therapy. Methods & clinical development · 2025Article
- Safety and efficacy of AAV8-aflibercept in treating choroidal neovascularization via single-cell RNA sequencing.Molecular therapy. Methods & clinical development · 2025Article
- How Close Are We to Achieving Durable and Efficacious Gene Therapy for Hemophilia A and B?Genes · 2025Review
- New hope for older SMA patients with next-generation self-complementary AAV gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Gene therapy with covalently closed-end AAV vector for spinal muscular atrophy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Orthogonal approaches to AAV vector characterization: Validating quantitative TEM for partially filled particles.Molecular therapy. Methods & clinical development · 2025Article
- AAV vector development, back to the future.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
Corrections and comments
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Authors and funding
12 authors at 5 institutions in 1 country.
Funding
Abstract
Self-complementary AAV vectors (scAAV) use a mutant inverted terminal repeat (mITR) for efficient packaging of complementary stranded DNA, enabling rapid transgene expression. However, inefficient resolution at the mITR leads to the packaging of monomeric or subgenomic AAV genomes. These noncanonical particles reduce transgene expression and may affect the safety of gene transfer. To address these issues, we have developed a novel class of scAAV vectors called covalently closed-end double-stranded AAV (cceAAV) that eliminate the mITR resolution step during production. Instead of using a mutant ITR, we used a 56-bp recognition sequence of protelomerase (TelN) to covalently join the top and bottom strands, allowing the vector to be generated with just a single ITR. To produce cceAAV vectors, the vector plasmid is initially digested with TelN, purified, and then subjected to a standard triple-plasmid transfection protocol followed by traditional AAV vector purification procedures. Such cceAAV vectors demonstrate yields comparable to scAAV vectors. Notably, we observed enhanced transgene expression as compared to traditional scAAV vectors. The treatment of mice with hemophilia B with cceAAV-FIX resulted in significantly enhanced long-term FIX expression. The cceAAV vectors hold several advantages over scAAV vectors, potentially leading to the development of improved human gene therapy drugs.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.