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Virus treatment questioned after gene therapy death
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“Although viral vectors have been widely used in the delivery of the CRISPR/Cas9 system in vitro and in vivo, their fundamental shortcomings, such as the risk of carcinogenesis, limited insertion size, immune responses and difficulty in large-scale production, severely limit their further applications.”
From Non-viral delivery systems for CRISPR/Cas9-based genome editing: challenges and opportunities (2018) · cites this paper as [69]
In the citing paper
“Zuris et al. injected Cas9:sgRNA complexes targeting GFP gene into the cochlea of transgenic Atoh1-GFP mice. This system resulted in ~20% Cas9-mediated genome modification in hair cells [69].”
From Non-viral delivery systems for CRISPR/Cas9-based genome editing: challenges and opportunities (2018) · cites this paper as [69]
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Pandey, S., Garg, P., Lim, K., Kim, J., Choung, Y., Choi, Y., Choung, P., Cho, C., & Chung, J. H. (2013) · Biomaterials · doi:10.1016/j.biomaterials.2013.01.098 · Cited by 39
“Introduction Non-viral gene delivery systems are superior to highly-efficient viral methods in a way being safer as they do not induce unwanted immune responses or aberrant gene expressions [1,2].”
BackgroundAdenovirus: a blueprint for non-viral gene delivery
Chailertvanitkul, V., & Pouton, C. W. (2010) · Current Opinion in Biotechnology · doi:10.1016/j.copbio.2010.06.011 · Cited by 39
“The Adv virion consists of an outer icosahedral capsid surrounded by an inner nucleoprotein core as illustrated in Figure 1 [1,2].”
BackgroundPotta, T., Zhen, Z., Grandhi, T. S. P., Christensen, M. D., Ramos, J., Breneman, C. M., & Rege, K. (2013) · Biomaterials · doi:10.1016/j.biomaterials.2013.10.069 · Cited by 38
Stachowiak, E. K., Roy, I., Lee, Y., Capacchietti, M., Aletta, J. M., Prasad, P. N., & Stachowiak, M. K. (2009) · Integrative Biology · doi:10.1039/b902617g · Cited by 38
Lee, M. R., & Jayant, R. D. (2018) · Cell and Tissue Research · doi:10.1007/s00441-018-2959-y · Cited by 39
“Non-viral gene delivery is an alternative approach that avoids the limitations and risks associated with delivering DNA to a cell using viral vectors (Lehrman 1999; Liu and Muruve 2003).”
Poly(amidoamine) Dendrimer-Gold Nanohybrids in Cancer Gene Therapy: A Concise Overview
Li, J., Shen, M., & Shi, X. (2020) · ACS Applied Bio Materials · doi:10.1021/acsabm.0c00863 · Cited by 37
Incani, V., Lin, X., Lavasanifar, A., & Uludağ, H. (2009) · ACS Applied Materials & Interfaces · doi:10.1021/am8002445 · Cited by 38
Terazaki, Y., 矢野, 正., Yuge, K., et al. (2003) · Hepatology · doi:10.1053/jhep.2003.50018 · Cited by 38
Cell‐selective viral gene delivery vectors for the vasculature
Baker, A. H., Kritz, A., Work, L. M., & Nicklin, S. A. (2004) · Experimental Physiology · doi:10.1113/expphysiol.2004.028126 · Cited by 38
“Email: ab11f@clinmed.gla.ac.uk Many setbacks have hit the gene therapy community in recent years, not least the death of Jesse Gelsinger (Lehrman, 1999) and the development of leukaemia in two patients on the severe combined immune deficiency syndrome (SCID) trials in France (Hacein-bey-abina et…”
BackgroundAngiogenic and antiangiogenic gene therapy
Małecki, M., Kołsut, P., & Proczka, R. M. (2005) · Gene Therapy · doi:10.1038/sj.gt.3302621 · Cited by 37
Gene therapy: principles and applications to hematopoietic cells
Tendeloo, V. F. V., Broeckhoven, C. V., & Berneman, Z. N. (2001) · Leukemia · doi:10.1038/sj.leu.2402085 · Cited by 37
Treatment to cure: Advancing AAV gene therapy manufacture
Young, P. M. (2023) · Drug Discovery Today · doi:10.1016/j.drudis.2023.103610 · Cited by 38
Polymeric nano-carriers for on-demand delivery of genes via specific responses to stimuli
Muhammad, K., Zhao, J., Gao, B., & Feng, Y. (2020) · Journal of Materials Chemistry B · doi:10.1039/d0tb01675f · Cited by 36
Targeted polymers for gene delivery
Hughes, J. A., & Rao, G. A. (2005) · Expert Opinion on Drug Delivery · doi:10.1517/17425247.2.1.145 · Cited by 36
“These concerns have been highlighted by cases of toxicity and mutagenecity observed in patients treated with viral vectors [11,12].”
BackgroundDesign of expanded bed supports for the recovery of plasmid DNA by anion exchange adsorption
Theodossiou, I., Søndergaard, M., & Thomas, O. R. T. (2001) · Bioseparation · doi:10.1023/a:1012078605874 · Cited by 36
Clinical trials of gene therapy for atherosclerotic cardiovascular disease
Freedman, S. B. (2002) · Current Opinion in Lipidology · doi:10.1097/00041433-200212000-00009 · Cited by 36
McCarthy, H. O., Coulter, J. A., Robson, T., & Hirst, D. G. (2008) · Journal of Pharmacy and Pharmacology · doi:10.1211/jpp.60.8.0007 · Cited by 34
“Elevated NO. levels confer desirable physiological responses that promote wound healing such as angiogenesis and the proliferation and migration of endothelial and epithelial cells (Leibovich et al 1994; Ziche et al 1994; Benrath et al 1995; Morbidelli et al 1996; Noiri et al 1996).”
BackgroundBartolami, E., Bessin, Y., Bettache, N., Gary‐Bobo, M., Garcia, M., Dumy, P., & Ulrich, S. (2015) · Organic & Biomolecular Chemistry · doi:10.1039/c5ob01404b · Cited by 39
Virus-mediated gene transfer to induce therapeutic angiogenesis: where do we stand?
Giacca, M. (2007) · PubMed Central · Cited by 34
Gene therapy for neurodegenerative and ocular diseases using lentiviral vectors
Ralph, G. S., Binley, K., Wong, L., Azzouz, M., & Mazarakis, N. D. (2005) · Clinical Science · doi:10.1042/cs20050158 · Cited by 35
Xia, J., Chen, L., Chen, J., Tian, H., Li, F., Zhu, X., Li, G., & Chen, X. (2010) · Macromolecular Bioscience · doi:10.1002/mabi.201000302 · Cited by 35
Kwon, J. S., Park, I., Cho, A. S., Shin, S. M., Hong, M. H., Jeong, S. Y., Kim, Y. S., Min, J.-J., Jeong, M. H., Kim, W. J., et al. (2009) · Journal of Controlled Release · doi:10.1016/j.jconrel.2009.05.023 · Cited by 34
Sunfish Amphiphiles: Conceptually New Carriers for DNA Delivery
Hulst, R., Muizebelt, I., Oosting, P., et al. (2004) · European Journal of Organic Chemistry · doi:10.1002/ejoc.200300524 · Cited by 34
Gürünlüoğlu, R., Özer, K., Skugor, B., et al. (2002) · Annals of Plastic Surgery · doi:10.1097/00000637-200208000-00009 · Cited by 34
Liu, J., Guo, S., Li, Z., Liu, L., & Gu, J. (2009) · Colloids and Surfaces B Biointerfaces · doi:10.1016/j.colsurfb.2009.04.026 · Cited by 33
Interaction between DNA and cationic diester-bonded Gemini surfactants
Yaseen, Z., Rehman, S. U., Tabish, M., & KABIR‐UD‐DIN, K. (2014) · Journal of Molecular Liquids · doi:10.1016/j.molliq.2014.05.013 · Cited by 33
Gene therapy with adenovirus-mediated VEGF enhances skin flap prefabrication
Gürünlüoğlu, R., Meirer, R., Shafighi, M., et al. (2005) · Microsurgery · doi:10.1002/micr.20142 · Cited by 33
Gene Therapy for Genetic Syndromes: Understanding the Current State to Guide Future Care
Henderson, M. L., Zieba, J. K., Li, X., Campbell, D. B., Williams, M. R., Vogt, D. L., Bupp, C. P., Edgerly, Y. M., Rajasekaran, S., Hartog, N., et al. (2024) · BioTech · doi:10.3390/biotech13010001 · Cited by 35
“One of the participants suffered from lethal systemic inflammation four days post-treatment [102].”
BackgroundInterleukin-8 gene silencing on pancreatic cancer cells using biodegradable polymer nanoplexes
Lin, G., Yang, C., Hu, R., Chen, C., Law, W., Anderson, T., Zhang, B., Nguyen, Q. T., Toh, H. T., Yoon, H. S., et al. (2014) · Biomaterials Science · doi:10.1039/c3bm60325c · Cited by 32
Zhang, L., Li, Y., Yu, J. C., Chen, Y. Y., & Chan, K. M. (2014) · Journal of Materials Chemistry B · doi:10.1039/c4tb01577k · Cited by 32
Sun, Y., Lv, X., Ding, P., Wang, L., Sun, Y., Li, S., Zhang, H., & Gao, Z. (2019) · Acta Biomaterialia · doi:10.1016/j.actbio.2019.06.059 · Cited by 32
Gene-based cancer vaccines: an ex vivo approach
Tendeloo, V. F. V., Broeckhoven, C. V., & Berneman, Z. N. (2001) · Leukemia · doi:10.1038/sj.leu.2402069 · Cited by 33
Whole-Heart Tissue Engineering and Cardiac Patches: Challenges and Promises
Akbarzadeh, A., Sobhani, S., Khaboushan, A. S., & Kajbafzadeh, A. M. (2023) · Bioengineering · doi:10.3390/bioengineering10010106 · Cited by 32
“So, the dose of vector that can be injected is limited [11].”
BackgroundDuan, Y., Guan, X., Ge, J., Quan, D., Zhuo, Y., Ye, H., & Shao, T. (2008) · PubMed Central · Cited by 34
Cheng, H. L., Zhu, J., Sun, Y., Cheng, S., Zhang, X., & Zhuo, R. (2008) · Bioconjugate Chemistry · doi:10.1021/bc700478s · Cited by 31
Drug Delivery Strategies to Overcome the Blood–Brain Barrier (BBB)
Helms, H. C. C., Kristensen, M., Saaby, L., Fricker, G., & Brodin, B. (2020) · Handbook of experimental pharmacology · doi:10.1007/164_2020_403 · Cited by 30
Li, Y., Tian, H., Ding, J., Dong, X., Chen, J., & Chen, X. (2014) · Polymer Chemistry · doi:10.1039/c3py01781h · Cited by 31
Effect of chain length on transfection properties of spermine-based gemini surfactants
Castro, M. J. L., Griffiths, D., Patel, A. R., et al. (2004) · Organic & Biomolecular Chemistry · doi:10.1039/b410240a · Cited by 31
Liposome-Mediated Suicide Gene Therapy in Humans
Reszka, R. C., Jacobs, A., & Voges, J. (2005) · Methods in enzymology on CD-ROM/Methods in enzymology · doi:10.1016/s0076-6879(05)91012-4 · Cited by 31
Adenoviral Strategies for the Gene Therapy of Cancer
Relph, K., Harrington, K. J., & Pandha, H. (2005) · Seminars in Oncology · doi:10.1053/j.seminoncol.2005.09.001 · Cited by 31
Singh, J., Michel, D. L., Getson, H. M., et al. (2015) · Nanomedicine · doi:10.2217/nnm.14.123 · Cited by 30
Gene-Therapeutic Strategies Targeting Angiogenesis in Peripheral Artery Disease
Sanada, F., Taniyama, Y., Muratsu, J., et al. (2018) · Medicines · doi:10.3390/medicines5020031 · Cited by 30
“However, significant concerns regarding the safety of adenovirus gene delivery has been raised after an 18-year-old man with an inherited enzyme deficiency died 4 days after a genetically altered adenovirus vector injection into his liver intravascularly [62].”
BackgroundProgress of Gene Therapy in Cardiovascular Disease
Shimamura, M., Nakagami, H., Sanada, F., & Morishita, R. (2020) · HYPERTENSION · doi:10.1161/hypertensionaha.120.14478 · Cited by 20
de Jongh, D., Massey, E. K., Cronin, A. J., Schermer, M., & Bunnik, E. M. (2022) · Transplant International · doi:10.3389/ti.2022.10751 · Cited by 35
“Such trials can lead to unforeseeable serious harm for research participants, as, for instance, has occurred during early-phase clinical trials of gene therapies in the 1990s, in which research participants have died (103).”
BackgroundHanda, M., Li, W., Morioka, K., et al. (2008) · Journal of Vascular Surgery · doi:10.1016/j.jvs.2008.07.029 · Cited by 30
Numerical optimization of gene electrotransfer into muscle tissue
Županič, A., Čorović, S., Miklavčič, D., et al. (2010) · BioMedical Engineering OnLine · doi:10.1186/1475-925x-9-66 · Cited by 30
“Currently, viral vectors boast the highest transfection efficiency, but this efficiency comes with an increased risk of viral infection [13,14].”
BackgroundGene therapy for the prevention of vein graft disease
Southerland, K. W., Frazier, S. B., Bowles, D. E., Milano, C. A., & Kontos, C. D. (2012) · Translational research · doi:10.1016/j.trsl.2012.12.003 · Cited by 29
Davies, J. B., Ciavatta, V. T., Boatright, J. H., & Nickerson, J. M. (2003) · PubMed · Cited by 30
Zhang, Z., Sha, X., Shen, A., et al. (2008) · Biochemical and Biophysical Research Communications · doi:10.1016/j.bbrc.2008.03.127 · Cited by 30
Mukherjee, K., Sen, J., & Chaudhuri, A. (2005) · FEBS Letters · doi:10.1016/j.febslet.2004.11.116 · Cited by 30
Gillard, M., Jia, Z., Hou, J. J. C., et al. (2014) · Biomacromolecules · doi:10.1021/bm5008376 · Cited by 29
Lee, M. C., Seonwoo, H., Jang, K. J., et al. (2021) · Bioactive Materials · doi:10.1016/j.bioactmat.2021.01.020 · Cited by 29
“responses or unwanted gene expression [11].”
Cell-Specific Targeting of Lipid-Based Carriers for ODN and DNA
Bartsch, M., Weeke-Klimp, A. H., Meijer, D. K. F., Scherphof, G. L., & Kamps, J. A. A. M. (2005) · Journal of Liposome Research · doi:10.1081/lpr-64961 · Cited by 30
“…of leukemia-like conditions in two children due to the employed viral vector have raised safety questions about gene therapy and in particular about viral vectors, which were used in 70% of the clinical trials (Hacein-Bey-Abina et al., 2003; Lehrman, 1999; Marshall, 1999; Raper et al., 2003).”
BackgroundRutanen, J., Markkanen, J. E., & Yl??-Herttuala, S. (2002) · Drugs · doi:10.2165/00003495-200262110-00001 · Cited by 29
“[77]”
From Nucleic Acids Sequences to Molecular Medicine
Erdmann, V. A., & Barciszewski, J. (2012) · RNA technologies · doi:10.1007/978-3-642-27426-8 · Cited by 28
Supramolecular complexes of DNA with cationic polymers: The effect of polymer concentration
Kondinskaia, D. A., & Gurtovenko, A. A. (2018) · Polymer · doi:10.1016/j.polymer.2018.03.048 · Cited by 28
“been of viral nature [1] and had therefore virus-specific safety issues such as high cytotoxicity and immunogenicity [2].”
Clérin, E., Marussig, M., Sahel, J., & Léveillard, T. (2020) · International Journal of Molecular Sciences · doi:10.3390/ijms21051625 · Cited by 31
Polyethylenimine-Mediated Cochlear Gene Transfer in Guinea Pigs
Tan, B. T. G., Foong, K. H., Lee, M. M. G., et al. (2008) · Archives of Otolaryngology - Head and Neck Surgery · doi:10.1001/archotol.134.8.884 · Cited by 28
Functional polymers of gene delivery for treatment of myocardial infarct
Won, Y.-W., Bull, D. A., & Kim, S. W. (2014) · Journal of Controlled Release · doi:10.1016/j.jconrel.2014.07.041 · Cited by 27
Pan, G., Shawer, M., Øie, S., & Lu, D. R. (2003) · Pharmaceutical Research · doi:10.1023/a:1023477317668 · Cited by 27
Shichiri, M., Tanaka, A., & Hirata, Y. (2003) · Gene Therapy · doi:10.1038/sj.gt.3301953 · Cited by 27
Nonviral Vector-Based Gene Transfection of Primary Human Skeletal Myoblasts
Ye, L., Haider, H. K., Esa, W. B., Law, P. K., Zhang, W., Su, L., Zhang, Y., & Sim, E. K. W. (2007) · Experimental Biology and Medicine · doi:10.3181/0706-rm-175 · Cited by 27
“However, problems such as high immunogenicity, tumorigenic potential, and inflammation inducement have limited the clinical application of viral vectors (7).”
BackgroundBioinspired peptides as versatile nucleic acid delivery platforms
Gopal, V. (2013) · Journal of Controlled Release · doi:10.1016/j.jconrel.2013.02.021 · Cited by 27
Ou, M., Kim, T., Yockman, J. W., et al. (2009) · Journal of Controlled Release · doi:10.1016/j.jconrel.2009.09.021 · Cited by 26
“While viral vectors, including adenovirus, adeno-associated virus, and retrevirus, have high gene transfection efficiency, they have limited clinical application due to their inherent potential for immunognicity, tumorigenicity, induction of an inflammatory response, and integration into the host genome[1,21,22].”
BackgroundSingh, R. S., & Chaudhuri, A. (2003) · FEBS Letters · doi:10.1016/s0014-5793(03)01381-4 · Cited by 26
Chen, D., Yuan, P., Tang, G., et al. (2010) · Soft Matter · doi:10.1039/b918966a · Cited by 26
Relph, K., Harrington, K. J., & Pandha, H. S. (2004) · BMJ · doi:10.1136/bmj.329.7470.839 · Cited by 26
Preventing saphenous vein graft failure: does gene therapy have a role?
Akowuah, E., Sheridan, P. J., Cooper, G. J., & Newman, C. M. H. (2003) · The Annals of Thoracic Surgery · doi:10.1016/s0003-4975(03)00505-8 · Cited by 25
Stem cells: the next therapeutic frontier.
Humes, H. D. (2005) · PubMed Central · Cited by 26
Blockade of Apoptosis as a Rational Therapeutic Strategy for the Treatment of Sepsis
Ayala, A., Wesche‐Soldato, D. E., Perl, M., Lomas‐Neira, J., Swan, R. Z., & Chung, C. (2006) · Novartis Foundation symposium · doi:10.1002/9780470059593.ch4 · Cited by 27
“This idea may also be an alternative to gene therapy, which, in the past, has not done well in the clinical setting due to marked inflammatory sequelae and oncogenesis (Lehrman 1999).”
BackgroundÖzenver, N., & Efferth, T. (2020) · Biochemical Pharmacology · doi:10.1016/j.bcp.2020.113792 · Cited by 24
The evolving role of medical geneticists in the era of gene therapy: An urgency to prepare
Vockley, J. E., Brunetti‐Pierri, N., Chung, W. K., et al. (2023) · Genetics in Medicine · doi:10.1016/j.gim.2023.100022 · Cited by 25
“Throughout that period, gene therapy for rare genetic disorders was often touted as being just around the corner; however, early gene therapy trials encountered serious safety issues that resulted in substantial delays.(5,6)”
Wang, B., Yi, W.-J., Zhang, J., et al. (2014) · Bioorganic & Medicinal Chemistry Letters · doi:10.1016/j.bmcl.2014.02.034 · Cited by 25
RNA‐Interferenz: von den Grundlagen zur therapeutischen Anwendung
Kurreck, J. (2009) · Angewandte Chemie · doi:10.1002/ange.200802092 · Cited by 26
DNA as therapeutics; an update
Saraswat, P., Soni, R., Bhandari, A., & Nagori, B. (2009) · Indian Journal of Pharmaceutical Sciences · doi:10.4103/0250-474x.58169 · Cited by 28
“Gelsinger developed a fever and blood clots throughout his body within hours of treatment to correct partial ornithine transcarbamylase (OTC) defi ciency, a rare metabolic disease that can cause a dangerous build-up of ammonia in the body and died four days later[7].”
BackgroundMarjanovič, I., Haberl, S., Miklavčič, D., et al. (2010) · The Journal of Membrane Biology · doi:10.1007/s00232-010-9282-1 · Cited by 25
“In gene therapy, DNA or RNA molecules are transferred into living cells in order to change their biological function for therapeutic purposes (Cavazzana-Calvo et al. 2000, 2004; Lehrman 1999; Hacein-Bey-Abina et al. 2003).”
BackgroundGelatin nanoparticles enhance delivery of hepatitis C virus recombinant NS2 gene
Sabet, S., George, M. A., El-Shorbagy, H. M., Bassiony, H., Farroh, K. Y., Youssef, T., & Salaheldin, T. A. (2017) · PLoS ONE · doi:10.1371/journal.pone.0181723 · Cited by 25
Clinical and Translational Landscape of Viral Gene Therapies
Yudaeva, A., Kostyusheva, A., Kachanov, A., Brezgin, S., Ponomareva, N., Parodi, A., Pokrovsky, V. S., Lukashev, A. N., Chulanov, V., & Kostyushev, D. S. (2024) · Cells · doi:10.3390/cells13221916 · Cited by 30
Cyclodextrin-based delivery systems for chemical and genetic drugs: Current status and future
Gao, Y., Sun, L., Qiao, C., Liu, Y., Wang, Y., Feng, R., Zhang, H., & Zhang, Y. (2024) · Carbohydrate Polymers · doi:10.1016/j.carbpol.2024.123174 · Cited by 28
Fu, A., Zhang, M., Gao, F., Xu, X., & Chen, Z. (2013) · PLoS ONE · doi:10.1371/journal.pone.0059642 · Cited by 28
Steele, T. W. J., & Shier, W. T. (2010) · Pharmaceutical Research · doi:10.1007/s11095-010-0058-1 · Cited by 24
“Viral vectors have had problems with immunogenicity (2,3) and carcinogenicity resulting from insertional mutagenicity (4,5).”
BackgroundLi, L., Yang, J., Wang, W., Yao, Y.-C., Fang, S., Dai, Z., Hong, H., Yang, X., Shuai, X., & Gao, G. (2012) · International Journal of Pharmaceutics · doi:10.1016/j.ijpharm.2012.08.043 · Cited by 24
Transplantation of Amniotic Epithelial Cells into Fetal Rat Liver by In Utero Manipulation
Takahashi, N., Enosawa, S., Mitani, T., et al. (2002) · Cell Transplantation · doi:10.3727/000000002783985602 · Cited by 24
Reproductive tract gene transfer
Daftary, G. S., & Taylor, H. S. (2003) · Fertility and Sterility · doi:10.1016/s0015-0282(03)00970-1 · Cited by 24
A Serum‐Tolerant Hydroxyl‐Modified Polyethylenimine as Versatile Carriers of pDNA/siRNA
Dong, X., Lın, L., Chen, J., et al. (2013) · Macromolecular Bioscience · doi:10.1002/mabi.201200346 · Cited by 23
Fujihara, Y., Koyama, H., Ohba, M., et al. (2008) · Wound Repair and Regeneration · doi:10.1111/j.1524-475x.2007.00339.x · Cited by 23
“Especially in gene therapy using viral vectors, the safety issue has not yet been completely resolved, blocking clinical application of gene therapy.(26) Furthermore, another crucial issue could be that delicate control of gene expression is difficult in the present state of gene therapy.”
BackgroundThe deLIVERed promises of gene therapy: Past, present, and future of liver-directed gene therapy
Puzzo, F., & Kay, M. A. (2025) · Molecular Therapy · doi:10.1016/j.ymthe.2025.03.041 · Cited by 26
“44,70 Due to their non-pathogenic nature and inherent liver uptake and tropism, AAV vectors are currently the preferred vehicle to deliver therapeutic genes for a broad spectrum of diseases.”
Cao, L., Yu, Y.-F., Li, Z., Zhong, N., Wang, G.-R., Xiao, Y., Liu, B., Wu, Q., Feng, C., & Bu, L. (2024) · Journal of Nanobiotechnology · doi:10.1186/s12951-024-02590-6 · Cited by 28
“The enthusiasm for viral vector research has largely waned after two clinical trials in which the use of viral vectors resulted in the deaths of volunteers [58, 59].”
BackgroundSuccessful liver-directed gene delivery by ERCP-guided hydrodynamic injection (with videos)
Kumbhari, V., Li, L., Piontek, K., Ishida, M., Fu, R., Khalil, B., Garrett, C. M., Liapi, E. A., Kalloo, A. N., & Selaru, F. M. (2018) · Gastrointestinal Endoscopy · doi:10.1016/j.gie.2018.06.022 · Cited by 23
“The specific objectives of this study were to (1) ascertain the parameters necessary for intrabiliary-delivered hydrodynamic gene delivery; (2) demonstrate feasibility of liver cell transduction using ERCP; and (3) assess whether successful transduction results in stable expression of the delivered plasmid proteins.”
BackgroundWan, C., Qian, J., Li, F., & Li, H. (2015) · Molecular Medicine Reports · doi:10.3892/mmr.2015.3703 · Cited by 22
“Although the use of a viral vector is able to provide a high transfer efficiency, the limited payload capacity, difficult large scale production and safety issues, such as potential immunogenicity and oncogenesis may hamper their clinical application (3-5).”
Orthopaedic applications of gene therapy
Lind, M., & Bünger, C. E. (2005) · International Orthopaedics · doi:10.1007/s00264-005-0650-x · Cited by 22
“However, they do not infect non-dividing cells, and one case of death during a clinical trial has been reported [11], these viruses probably have limited usage in future clinical practice.”
A mechanistic explanation of the inhibitory role of the protein corona on liposomal gene expression
Palchetti, S., Digiacomo, L., Giulimondi, F., Pozzi, D., Peruzzi, G., Ferri, G., Amenitsch, H. W., Cardarelli, F., Mahmoudi, M., & Caracciolo, G. (2019) · Biochimica et Biophysica Acta (BBA) - Biomembranes · doi:10.1016/j.bbamem.2019.183159 · Cited by 21
Perivascular administration of drugs and genes as a means of reducing vein graft failure
Wiedemann, D., Kocher, A. A., Bonaros, N. E., et al. (2012) · Current Opinion in Pharmacology · doi:10.1016/j.coph.2012.02.012 · Cited by 21
“The death of a patient during an adenoviral clinical trial in the US raised additional concerns [80,95].”
BackgroundA small molecule for theraNOstic targeting of cancer cells
Ravikumar, G., Bagheri, M., Saini, D. K., et al. (2017) · Chemical Communications · doi:10.1039/c7cc08526e · Cited by 21
Han, Y.-F., Han, Y.-Q., Pan, Y., Chen, Y.-L., & Chai, J.-K. (2010) · Transplantation Proceedings · doi:10.1016/j.transproceed.2009.12.070 · Cited by 21
Frank, S., Gauster, M., Bogner‐Strauß, J. G., et al. (2001) · Gene Therapy · doi:10.1038/sj.gt.3301434 · Cited by 21
Recombinant Vectors as Influenza Vaccines
Kopecky-Bromberg, S. A., & Palese, P. (2009) · Current topics in microbiology and immunology · doi:10.1007/978-3-540-92165-3_13 · Cited by 20
“Adenovirus vectors have been used in gene therapy trials as well as for therapy against cancer, and it was during a gene therapy trial that a participant died of an inflammatory response after receiving a high dose of vector (3.8 × 1013 virus particles) (Lehrman 1999; Marshall 1999).”
MethodInfluentialNanocarrier Mediated siRNA Delivery Targeting Stem Cell Differentiation
Fernandes, F., Kotharkar, P., Chakravorty, A., Kowshik, M., & Talukdar, I. (2019) · Current Stem Cell Research & Therapy · doi:10.2174/1574888x14666191202095041 · Cited by 20
TERT promoter-driven adenovirus vector for cancer gene therapy via systemic injection
Yao, X., Yoshioka, Y., Eto, Y., et al. (2007) · Biochemical and Biophysical Research Communications · doi:10.1016/j.bbrc.2007.08.001 · Cited by 20
Targeted gene therapy: frontiers in the development of 'smart drugs'.
Tarner, I., Müller-Ladner, U., & Fathman, C. (2004) · Trends in Biotechnology · doi:10.1016/j.tibtech.2004.04.001 · Cited by 19
Metadata
- Authors
- Lehrman, S.
- Corresponding author
- Lehrman, Sally
- ORCID
- Lehrman, Sally
- Venue
- Nature
- Published
- 1999-10-07
- Volume
- 401
- Issue
- 6753
- Pages
- 517-518
- ISSN
- 0028-0836 (ISSN-L)
- 1476-4687
- OpenAlex
- W1606853210
- Cited by
- 576
- References
- 0
- Citations by year
- 2026: 6 · 2025: 9 · 2024: 11 · 2023: 15 · 2022: 17 · 2021: 12 · 2020: 18 · 2019: 16 · 2018: 18 · 2017: 14 · 2016: 14 · 2015: 24 · 2014: 30 · 2013: 23 · 2012: 31
- Type
- other
- Language
- en
- Open access
- closed
- Licence
- Locations
- Nature (publishedVersion)
- PubMed (publishedVersion)
- RePEc: Research Papers in Economics (submittedVersion)
- DigitalGeorgetown (Georgetown University Library) (submittedVersion)
- Funders
- Brigham and Women's Hospital
- University of East Anglia
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Health Sciences › Nursing › Issues, ethics and legal aspects › Biomedical Ethics and Regulation
- Health Sciences › Medicine › Public Health, Environmental and Occupational Health › Ethics in Clinical Research
- Keywords
- gene therapy, viral vectors, recombinant adeno-associated virus, clinical trial safety
- MeSH terms
- Adenoviridae, Adult, Fever, Genetic Vectors, Humans, Male, Ornithine Carbamoyltransferase, Thrombosis, Genetic Therapy, Clinical Trials, Phase I as Topic, Fatal Outcome, Risk Assessment, Ornithine Carbamoyltransferase Deficiency Disease, Advisory Committees, Government Regulation, Federal Government, Research Subjects
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