ArticleScientific reports2017
Lentiviral vectors can be used for full-length dystrophin gene therapy.
Article in Scientific reports, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 27 papers.
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Who cites it
27 citing papers in PubMed.
- Article
- Dual AAV vectors for efficient delivery of large transgenes.Nature protocols · 2026Review
- Genetic strategies for therapy of Duchenne muscular dystrophy.Molecular therapy. Nucleic acids · 2025Review
- Development of compact transcriptional effectors using high-throughput measurements in diverse contexts.Nature biotechnology · 2025Article
- Delivery of genetic medicines for muscular dystrophies.Cell reports. Medicine · 2025Review
- RNA Structure: Past, Future, and Gene Therapy Applications.International journal of molecular sciences · 2024Review
- Exploring Advanced CRISPR Delivery Technologies for Therapeutic Genome Editing.Small science · 2024Article
- Designing molecules: directing stem cell differentiation.Frontiers in bioengineering and biotechnology · 2024Review
- The double-edge sword of CRISPR application forOncotarget · 2023Article
- Delivery challenges for CRISPR-Cas9 genome editing for Duchenne muscular dystrophy.Biophysics reviews · 2023Review
- Transplantation of Adipose-Tissue-Engineered Constructs with CRISPR-Mediated UCP1 Activation.International journal of molecular sciences · 2023Article
- Application of CRISPR for In Vivo Mouse Cancer Studies.Cancers · 2022Review
- Overview of Neutralization Assays and International Standard for Detecting SARS-CoV-2 Neutralizing Antibody.Viruses · 2022Review
- Optimized lentiviral vector to restore full-length dystrophin via a cell-mediated approach in a mouse model of Duchenne muscular dystrophy.Molecular therapy. Methods & clinical development · 2022Article
- Viruses as tools in gene therapy, vaccine development, and cancer treatment.Archives of virology · 2022Review
- The role of gap junctions in cell death and neuromodulation in the retina.Neural regeneration research · 2021Review
- Changes in Myonuclear Number During Postnatal Growth - Implications for AAV Gene Therapy for Muscular Dystrophy.Journal of neuromuscular diseases · 2021Review
- Modified mRNA as a Therapeutic Tool for the Heart.Cardiovascular drugs and therapy · 2020Review
- Gene therapy for inherited metabolic diseases.Journal of mother and child · 2020Review
- The delivery challenge: fulfilling the promise of therapeutic genome editing.Nature biotechnology · 2020Review
Corrections and comments
- Erratum issued
Authors and funding
11 authors.
Funding
Abstract
Duchenne Muscular Dystrophy (DMD) is caused by a lack of dystrophin expression in patient muscle fibres. Current DMD gene therapy strategies rely on the expression of internally deleted forms of dystrophin, missing important functional domains. Viral gene transfer of full-length dystrophin could restore wild-type functionality, although this approach is restricted by the limited capacity of recombinant viral vectors. Lentiviral vectors can package larger transgenes than adeno-associated viruses, yet lentiviral vectors remain largely unexplored for full-length dystrophin delivery. In our work, we have demonstrated that lentiviral vectors can package and deliver inserts of a similar size to dystrophin. We report a novel approach for delivering large transgenes in lentiviruses, in which we demonstrate proof-of-concept for a 'template-switching' lentiviral vector that harnesses recombination events during reverse-transcription. During this work, we discovered that a standard, unmodified lentiviral vector was efficient in delivering full-length dystrophin to target cells, within a total genomic load of more than 15,000 base pairs. We have demonstrated gene therapy with this vector by restoring dystrophin expression in DMD myoblasts, where dystrophin was expressed at the sarcolemma of myotubes after myogenic differentiation. Ultimately, our work demonstrates proof-of-concept that lentiviruses can be used for permanent full-length dystrophin gene therapy, which presents a significant advancement in developing an effective treatment for DMD.
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