The DOI matches its Crossref record; 164 of the 164 references it cites were found in a registry or an index.
GENE THERAPY: Twenty-First Century Medicine
Abstract
▪ Abstract Broadly defined, the concept of gene therapy involves the transfer of genetic material into a cell, tissue, or whole organ, with the goal of curing a disease or at least improving the clinical status of a patient. A key factor in the success of gene therapy is the development of delivery systems that are capable of efficient gene transfer in a variety of tissues, without causing any associated pathogenic effects. Vectors based upon many different viral systems, including retroviruses, lentiviruses, adenoviruses, and adeno-associated viruses, currently offer the best choice for efficient gene delivery. Their performance and pathogenicity has been evaluated in animal models, and encouraging results form the basis for clinical trials to treat genetic disorders and acquired diseases. Despite some initial success in these trials, vector development remains a seminal concern for improved gene therapy technologies.
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Cites
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Cited by
Gene therapy clinical trials worldwide to 2017: An update
Ginn, S. L., Amaya, A. K., Alexander, I. E., Edelstein, M., & Abedi, M. R. (2018) · The Journal of Gene Medicine · doi:10.1002/jgm.3015 · Cited by 903
Diseases Caused by Defects in the Visual Cycle: Retinoids as Potential Therapeutic Agents
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Gene therapy for lysosomal storage diseases
Sands, M. S., & Davidson, B. L. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2006.01.006 · Cited by 285
A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice
Brown, B. D., Cantore, A., Annoni, A., Sergi, L. S., Lombardo, A., Della Valle, P., D'Angelo, A., & Naldini, L. (2007) · Blood · doi:10.1182/blood-2007-03-078493 · Cited by 252
DNA Shuffling of Adeno-associated Virus Yields Functionally Diverse Viral Progeny
Koerber, J. T., Jang, J.-H., & Schaffer, D. V. (2008) · Molecular Therapy · doi:10.1038/mt.2008.167 · Cited by 181
Metadata
- Authors
- Verma, I. M., & Weitzman, M. D.
- Venue
- Annual Review of Biochemistry
- Publisher
- Annual Reviews
- Published
- 2005-06-01
- Volume
- 74
- Issue
- 1
- Pages
- 711-738
- ISSN
- 0066-4154 (ISSN-L)
- 1545-4509
- OpenAlex
- W2170533808
- Cited by
- 616
- References
- 164
- FWCI
- 3.70
- Type
- journal article
- Language
- en
- Open access
- closed
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