Research Highlight |
Featured
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In Brief |
Towards gene therapy for Tay-Sachs disease
Flotte et al. describe the first test of adeno-associated virus-based gene therapy for Tay-Sachs disease in humans. Delivery to the thalamus and cerebrospinal fluid was found to be broadly safe, providing a firm basis for future clinical trials.
- Dorothy Clyde
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In Brief |
Fighting fibrosis with transient CAR T cells
A report in Science describes a method to generate transient chimeric antigen receptor (CAR) T cells in vivo via lipid nanoparticle-mediated T cell-targeted delivery of a CAR-encoding nucleoside-modified mRNA.
- Dorothy Clyde
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Review Article |
Engineering adeno-associated virus vectors for gene therapy
Adeno-associated virus (AAV) vector-mediated gene delivery has had long-term therapeutic effects for several diseases, including haemophilia and Duchenne muscular dystrophy. Genetically modifying AAV vectors to increase their transduction efficiency, vector tropism and ability to avoid the host immune response may further increase the success of AAV gene therapy.
- Chengwen Li
- & R. Jude Samulski
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Research Highlight |
Gold rush to gene-editing in the brain
A study in Nature Biomedical Engineering reports improvements in neurological symptoms in a mouse model of fragile X syndrome after non-viral delivery of Cas9 ribonucleoproteins to the brain.
- Michelle Trenkmann
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Review Article |
Non-viral vectors for gene-based therapy
This Review introduces the biological barriers to gene deliveryin vivoand discusses recent advances in material sciences, nanotechnology and nucleic acid chemistry that have yielded promising non-viral systems for the delivery of DNA, mRNA, small interfering RNAs and microRNAs, some of which are currently undergoing testing in clinical trials.
- Hao Yin
- , Rosemary L. Kanasty
- & Daniel G. Anderson
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Progress |
Engineering adeno-associated viruses for clinical gene therapy
Although gene delivery vectors based on adeno-associated viruses (AAVs) have emerged as safe and effective for numerous clinical gene therapy applications, many challenges remain. Recent advances in AAV vector development through rational design and directed evolution, as well as in the design of novel genetic cargoes, promise to extend clinical successes of AAV-mediated gene therapy.
- Melissa A. Kotterman
- & David V. Schaffer
In vivo selection of gene-corrected cells
In vivo genome editing — growing in strength
Insulating from genotoxicity