The DOI matches its Crossref record; 7 of the 7 references it cites were found in a registry or an index.
Improved Method of Recombinant AAV2 Delivery for Systemic Targeted Gene Therapy
Open accessAbstract
A major hurdle in most current gene therapy modalities is the ability to transduce target tissues at very high efficiencies that ultimately lead to therapeutic levels of transgene expression. We have developed a novel method of recombinant adeno-associated virus 2 (rAAV) delivery that results in increased vector transduction efficiencies using microspheres reversibly conjugated to rAAV vectors. We hypothesize that conjugation to microspheres should result in a higher effective concentration of vector as well as longer relative exposure time of vector to target cells as it moves through the tissue vasculature. In vitro experiments demonstrate that the same level of transduction seen with free vector can be achieved using 1% of vector when conjugated to microspheres. In addition, using magnetic microspheres, the region of infection can be targeted. In vivo, we demonstrate that microsphere-mediated delivery of rAAV vector results in higher transduction efficiencies than delivery with free vector alone when administered either intramuscularly or intravenously. Furthermore, we demonstrate targeting of transgene expression to specific tissues by retention of microsphere-bound vector in the capillary bed. These studies demonstrate a novel method to deliver rAAV vectors more effectively that could prove to be a successful alternative mode of virus-mediated human gene therapy.
Overview
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Integrity signals
Registry
Cites
Statistics
Verification
Claims the paper makes
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Self-check
Percentages, sums and reported differences re-computed from the paper’s own numbers.
The self-check needs the paper’s full text.
Citations
Cites
Ferrari, F. K., Samulski, T., Shenk, T., & Samulski, R. J. (1996) · Journal of Virology · doi:10.1128/jvi.70.5.3227-3234.1996 · Cited by 761
Infectious entry pathway of adeno-associated virus and adeno-associated virus vectors
Bartlett, J. S., Wilcher, R., & Samulski, R. J. (2000) · Journal of Virology · doi:10.1128/jvi.74.6.2777-2785.2000 · Cited by 532
Duan, D., Sharma, P., Yang, J., Yue, Y., Dudus, L., Zhang, Y., Fisher, K. J., & Engelhardt, J. F. (1998) · Journal of Virology · doi:10.1128/jvi.72.11.8568-8577.1998 · Cited by 522
Chao, H., Liu, Y., Rabinowitz, J., Li, C., Samulski, R. J., & Walsh, C. E. (2000) · Molecular Therapy · doi:10.1006/mthe.2000.0219 · Cited by 366
Loeb, J. E., Cordier, W. S., Harris, M. E., Weitzman, M. D., & Hope, T. J. (1999) · Human Gene Therapy · doi:10.1089/10430349950016942 · Cited by 279
Insertional Mutagenesis of AAV2 Capsid and the Production of Recombinant Virus
Rabinowitz, J. E., Xiao, W., & Samulski, R. J. (1999) · Virology · doi:10.1006/viro.1999.0045 · Cited by 136
Structural Analysis of Adeno-Associated Virus Transduction Circular Intermediates
Duan, D., Yan, Z., Yue, Y., & Engelhardt, J. F. (1999) · Virology · doi:10.1006/viro.1999.9821 · Cited by 100 · cited 1×
“In addition, recent works describing the modes of entry into target cells [13–16], structural status of the rAAV genomes [17–20], the cell-cycle status of the infected cell [21,22], and the involvement of intracellular proteins in rAAV-mediated transgene expression [14,23–25] have provided a better understanding of th…”
Background
Cited by
Smart micro/nanoparticles in stimulus-responsive drug/gene delivery systems
Karimi, M., Ghasemi, A., Sahandi Zangabad, P., Rahighi, R., Moosavi Basri, S. M., Mirshekari, H., Amiri, M., Shafaei Pishabad, Z., Aslani, A., Bozorgomid, M., et al. (2016) · Chemical Society Reviews · doi:10.1039/c5cs00798d · Cited by 1,519
Progress in applications of magnetic nanoparticles in biomedicine
Pankhurst, Q. A., Thanh, N. T. K., Jones, S. K., & Dobson, J. (2009) · Journal of Physics D: Applied Physics · doi:10.1088/0022-3727/42/22/224001 · Cited by 1,455
Self-citationSynthesis, Functionalization, and Biomedical Applications of Multifunctional Magnetic Nanoparticles
Hao, R., Xing, R., Xu, Z., Hou, Y., Gao, S., & Sun, S. (2010) · Advanced Materials · doi:10.1002/adma.201000260 · Cited by 1,372
Magnetic Nanoparticles: Surface Effects and Properties Related to Biomedicine Applications
Issa, B., Obaidat, I., Albiss, B., & Haik, Y. (2013) · International Journal of Molecular Sciences · doi:10.3390/ijms141121266 · Cited by 1,245
Iron Oxide Nanoparticles for Magnetically-Guided and Magnetically-Responsive Drug Delivery
Estelrich, J., Escribano, E., Queralt, J., & Busquets, M. (2015) · International Journal of Molecular Sciences · doi:10.3390/ijms16048070 · Cited by 532
Metadata
- Authors
- Mah, C., Fraites, T. J., Zolotukhin, I., Song, S., Flotte, T. R., Dobson, J., Batich, C., & Byrne, B. J.
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2002-07
- Volume
- 6
- Issue
- 1
- Pages
- 106-112
- ISSN
- 1525-0016 (ISSN-L)
- OpenAlex
- W2062787494
- Cited by
- 178
- References
- 7
- FWCI
- 3.00
- Type
- journal article
- Language
- en
- Open access
- hybrid
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review
How to cite
Cite CoChat for the checks on this page, and the paper for its own findings.
- This page
- CoChat. (2026). Citation check: Improved Method of Recombinant AAV2 Delivery for Systemic Targeted Gene Therapy. https://app.cochat.ai/papers/10.1006/mthe.2001.0636
- The paper
- Mah, C., Fraites, T. J., Zolotukhin, I., Song, S., Flotte, T. R., Dobson, J., Batich, C., & Byrne, B. J. (2002). Improved Method of Recombinant AAV2 Delivery for Systemic Targeted Gene Therapy. Molecular Therapy. https://doi.org/10.1006/mthe.2001.0636
Data sources
Bibliographic data: Semantic Scholar, OpenAlex, Crossref.
Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
Semantic Scholar’s data is described in: Kinney, R., et al. (2023). The Semantic Scholar Open Data Platform. arXiv:2301.10140.
