ArticleMolecular therapy : the journal of the American Society of Gene Therapy2020
Effective and Accurate Gene Silencing by a Recombinant AAV-Compatible MicroRNA Scaffold.
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Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers.
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24citing papers in PubMed
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24 citing papers in PubMed, 33 citations in OpenAlex.
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- Impeding the NHEJ Pathway for Overcoming Radioresistance in the Context of Precision Radiotherapy of Cancer.Pharmaceutics · 2026Review
- shRNAI: A deep neural network for the design of highly potent shRNAs.Molecular therapy. Nucleic acids · 2025Article
- Preclinical Development of a Vectorized Artificial miRNA Gene Therapy for Tauopathies.bioRxiv : the preprint server for biology · 2025Article
- miR-375 protects against acetaminophen-induced acute liver failure by orchestrating pharmacogene expression.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Adeno-associated Virus-mediated Silencing of Sox4 Leads to Long-Term Amelioration of Liver Phenotypes in Mouse Models of Alagille Syndrome.Gastroenterology · 2025Article
- Engineered microRNA scaffolds for potent gene silencing in vivo.Scientific reports · 2025Article
- Gene therapy then and now: A look back at changes in the field over the past 25 years.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- A self-complementary AAV proviral plasmid that reduces cross-packaging and ITR promoter activity in AAV vector preparations.Molecular therapy. Methods & clinical development · 2024Article
- Engineering a targeted and safe bone anabolic gene therapy to treat osteoporosis in alveolar bone loss.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Conditional RNA interference in mammalian cells via RNA transactivation.Nature communications · 2024Article
- Sequencing-guided design of genetically encoded small RNAs targeting CAG repeats for selective inhibition of mutantMolecular therapy. Nucleic acids · 2024Article
- Efficacy and safety of a SOD1-targeting artificial miRNA delivered by AAV9 in mice are impacted by miRNA scaffold selection.Molecular therapy. Nucleic acids · 2023Article
- AAV-Mediated Targeting of the Activin A-ACVR1Biomolecules · 2023Article
- Schnurri-3 inhibition suppresses bone and joint damage in models of rheumatoid arthritis.Proceedings of the National Academy of Sciences of the United States of America · 2023Article
- RNAi-mediated rheostat for dynamic control of AAV-delivered transgenes.Nature communications · 2023Article
- AAV- based vector improvements unrelated to capsid protein modification.Frontiers in medicine · 2023Review
- Suppression of heterotopic ossification in fibrodysplasia ossificans progressiva using AAV gene delivery.Nature communications · 2022Article
- AAV-mediated delivery of osteoblast/osteoclast-regulating miRNAs for osteoporosis therapy.Molecular therapy. Nucleic acids · 2022Article
Corrections and comments
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Authors and funding
16 authors at 2 institutions in 2 countries.
Jun XieHorae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA; Viral Vector Core, University of Massachusetts Medical School, Worcester, MA, USA; Li Weibo Institute for Rare Diseases Research, University of Massachusetts Medical School, Worcester, MA, USA; Department of Microbiology and Physiological Systems, University of Massachusetts Medical School, Worcester, MA, USA. Electronic address: jun.xie@umassmed.edu.
Phillip W L TaiHorae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA; Li Weibo Institute for Rare Diseases Research, University of Massachusetts Medical School, Worcester, MA, USA; Department of Microbiology and Physiological Systems, University of Massachusetts Medical School, Worcester, MA, USA.
Alexander BrownHorae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA; Li Weibo Institute for Rare Diseases Research, University of Massachusetts Medical School, Worcester, MA, USA; Department of Microbiology and Physiological Systems, University of Massachusetts Medical School, Worcester, MA, USA.
Shoufang GongHorae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA; Li Weibo Institute for Rare Diseases Research, University of Massachusetts Medical School, Worcester, MA, USA; Department of Microbiology and Physiological Systems, University of Massachusetts Medical School, Worcester, MA, USA.
Sha ZhuHorae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA; Li Weibo Institute for Rare Diseases Research, University of Massachusetts Medical School, Worcester, MA, USA; Department of Microbiology and Physiological Systems, University of Massachusetts Medical School, Worcester, MA, USA.
Yi WangHorae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA; Li Weibo Institute for Rare Diseases Research, University of Massachusetts Medical School, Worcester, MA, USA; Department of Microbiology and Physiological Systems, University of Massachusetts Medical School, Worcester, MA, USA; Research Unit of Infection and Immunity, Department of Pathophysiology, West China College of Basic and Forensic Medicine, Sichuan University, Chengdu, Sichuan, People's Republic of China.
Chengjian LiFornax Biotech, Worcester, MA, USA.
Cansu ColpanRNA Therapeutics Institute and Howard Hughes Medical Institute, University of Massachusetts Medical School, Worcester, MA, USA.
Qin SuHorae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA; Viral Vector Core, University of Massachusetts Medical School, Worcester, MA, USA; Li Weibo Institute for Rare Diseases Research, University of Massachusetts Medical School, Worcester, MA, USA.
Ran HeHorae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA; Viral Vector Core, University of Massachusetts Medical School, Worcester, MA, USA; Li Weibo Institute for Rare Diseases Research, University of Massachusetts Medical School, Worcester, MA, USA.
Hong MaHorae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA; Viral Vector Core, University of Massachusetts Medical School, Worcester, MA, USA; Li Weibo Institute for Rare Diseases Research, University of Massachusetts Medical School, Worcester, MA, USA.
Jia LiHorae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA; Li Weibo Institute for Rare Diseases Research, University of Massachusetts Medical School, Worcester, MA, USA; Department of Microbiology and Physiological Systems, University of Massachusetts Medical School, Worcester, MA, USA.
Hanqing YeHorae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA; Li Weibo Institute for Rare Diseases Research, University of Massachusetts Medical School, Worcester, MA, USA; Department of Microbiology and Physiological Systems, University of Massachusetts Medical School, Worcester, MA, USA.
Jihye KoHorae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA; Viral Vector Core, University of Massachusetts Medical School, Worcester, MA, USA; Li Weibo Institute for Rare Diseases Research, University of Massachusetts Medical School, Worcester, MA, USA.
Phillip D ZamoreRNA Therapeutics Institute and Howard Hughes Medical Institute, University of Massachusetts Medical School, Worcester, MA, USA. Electronic address: phillip.zamore@umassmed.edu.
Guangping GaoHorae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA; Viral Vector Core, University of Massachusetts Medical School, Worcester, MA, USA; Li Weibo Institute for Rare Diseases Research, University of Massachusetts Medical School, Worcester, MA, USA; Department of Microbiology and Physiological Systems, University of Massachusetts Medical School, Worcester, MA, USA. Electronic address: guangping.gao@umassmed.edu.
University of Massachusetts Chan Medical School · USHoward Hughes Medical Institute · US
Funding
Viral escape from AAV expressed transgenesP01AI100263 · NIAID · SCRIPPS FLORIDA · PI GAO, GUANGPING · 2012 to 2016
$11.9MViral Vector CoreP01HL131471 · NHLBI · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI FLOTTE, TERENCE R. · 2016 to 2020
$11.3MOligodendrocyte-focused rAAV gene therapy strategies for Canavan disease and LeukodystrophiesR01NS076991 · NINDS · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI Guangping Gao · 2012 to 2026
$5.7MNext Generation of Recombinant AAV Serotype Vectors for Gene TherapyR01HL097088 · NHLBI · UNIVERSITY OF FLORIDA · PI GAO, GUANGPING, HERZOG, ROLAND W. · 2010 to 2018
$5.4MNHLBI NIH HHS P01 HL131471NHLBI NIH HHS R01 HL097088NIAID NIH HHS P01 AI100263NINDS NIH HHS R01 NS076991
Abstract
Short hairpin RNAs that are delivered by recombinant adeno-associated virus (rAAV) have the potential to elicit long-term RNAi therapy for human disease. However, the discovery that short hairpin sequences can cause truncation of the rAAV genome calls into question the efficiency and gene-silencing specificity of this strategy in humans. Here, we report that embedding the guide strand of a small silencing RNA into an artificial microRNA (miRNA) scaffold derived from mouse miRNA-33 ensures rAAV genomic integrity and reduces off-targeting by 10-fold, while maintaining effective in vivo target gene repression in mice.
Indexed as
Gene SilencingAnimalsDependovirusGenetic VectorsGenome, ViralHumansMiceMicroRNAsNucleic Acid ConformationRNA InterferenceRNA, Small InterferingRNA StabilityRNA, ViralMicroRNAsMIRN33a microRNA, humanRNA, Small InterferingRNA, Viral
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