The DOI matches its Crossref record; 241 of the 241 references it cites were found in a registry or an index.
Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality
Open accessAbstract
Gene transfer has long been a compelling yet elusive therapeutic modality. First mainly considered for rare inherited disorders, gene therapy may open treatment opportunities for more challenging and complex diseases such as Alzheimer’s or Parkinson’s disease. Today, examples of striking clinical proof of concept, the first gene therapy drugs coming onto the market, and the emergence of powerful new molecular tools have broadened the number of avenues to target neurological disorders but have also highlighted safety concerns and technology gaps. The vector of choice for many nervous system targets currently is the adeno-associated viral (AAV) vector due to its desirable safety profile and strong neuronal tropism. In aggregate, the clinical success, the preclinical potential, and the technological innovation have made therapeutic AAV drug development a reality, particularly for nervous system disorders. Here, we discuss the rationale, opportunities, limitations, and progress in clinical AAV gene therapy.
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Viral vector‐based gene therapies in the clinic
Zhao, Z., Anselmo, A. C., & Mitragotri, S. (2022) · Bioengineering & Translational Medicine · doi:10.1002/btm2.10258 · Cited by 269
Adeno-Associated Virus Toolkit to Target Diverse Brain Cells
Challis, R. C., Ravindra Kumar, S., Chen, X., Goertsen, D., Coughlin, G. M., Hori, A. M., Chuapoco, M. R., Otis, T. S., Miles, T. F., & Gradinaru, V. (2022) · Annual Review of Neuroscience · doi:10.1146/annurev-neuro-111020-100834 · Cited by 148
AAV vectors applied to the treatment of CNS disorders: Clinical status and challenges
Kang, L., Jin, S., Wang, J., Lv, Z., Xin, C., Tan, C., Zhao, M., Wang, L., & Liu, J. (2023) · Journal of Controlled Release · doi:10.1016/j.jconrel.2023.01.067 · Cited by 131
Yao, Y., Wang, J., Liu, Y., Qu, Y., Wang, K., Zhang, Y., Chang, Y., Yang, Z., Wan, J., Liu, J., et al. (2022) · Nature Biomedical Engineering · doi:10.1038/s41551-022-00938-7 · Cited by 120
Metadata
- Authors
- Hudry, E., & Vandenberghe, L. H.
- Venue
- Neuron
- Publisher
- Elsevier BV
- Published
- 2019-03
- Volume
- 101
- Issue
- 5
- Pages
- 839-862
- ISSN
- 0896-6273 (ISSN-L)
- OpenAlex
- W2921986557
- Cited by
- 369
- References
- 241
- FWCI
- 26.41
- Type
- journal article
- Language
- en
- Open access
- bronze
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Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
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