Adeno‐associated virus vectors and hematology

Russell, David W.; Kay, Mark A.

doi:10.1182/blood.v94.3.864.415k34_864_874

Cited by 104Virus-based gene therapy researchHemophilia Treatment and ResearchRNA Interference and Gene Delivery

Adeno‐associated virus vectors and hematology

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Russell, David W., Kay, Mark A.·1999·Blood

DOI 10.1182/blood.v94.3.864.415k34_864_874PMC PMC3739711

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Abstract

Gene therapists have a special fondness for hematology. Initially, this was due to the many inherited diseases that could potentially be cured by ex vivo genetic modification of hematopoietic stem cells. Once the early murine leukemia virus (MLV) vectors were found to transduce only a small percentage of primate stem cells, the search began for other types of vectors that might work more efficiently, and adeno-associated virus (AAV) vectors were among the first alternatives to be investigated. Although a substantial amount of work with AAV vectors has occurred over the last several years, the bulk of evidence suggests that they transduce hematopoietic stem cells poorly, and that they will not replace retroviral vectors in stem cell gene transfer applications. Instead, it is applications involving the production of secreted proteins that now appear especially promising for AAV vectors, where they are delivered by direct injection rather than ex vivo cell manipulation. Recent animal studies have shown that AAV vectors encoding factor IX can lead to long-term, therapeutic clotting factor levels after in vivo administration, setting the stage for what may well be a cure for hemophilia B in the near future. Animal experiments have also shown that AAV vectors expressing erythropoietin can produce dramatic increases in hematocrit levels. Here we will review the role of AAV vectors in the field of hematology, including the basic biology of AAV, vector transduction properties, their use in hematopoietic cells, and their potential for delivering secreted proteins such as clotting factors and cytokines.

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Gene addition approaches with AAV are limited by transient and unregulated expression.
“Despite many advances, gene addition approaches with adeno-associated virus (AAV) are limited by transient and unregulated expression,2”
From Adeno‐associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo†‡ (2010) · cites this paper as [2]

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Latent infection of KB cells with adeno-associated virus type 2
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Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics
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AAV vectors: is clinical success on the horizon?
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Viral vectors for gene delivery in tissue engineering☆
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Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors
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Local Adeno-Associated Virus-Mediated Interleukin 10 Gene Transfer Has Disease-Modifying Effects in a Murine Model of Sjögren's Syndrome
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Sustained Expression of Therapeutic Level of Factor IX in Hemophilia B Dogs by AAV-Mediated Gene Therapy in Liver
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Recruitment of Single-Stranded Recombinant Adeno-Associated Virus Vector Genomes and Intermolecular Recombination Are Responsible for Stable Transduction of Liver In Vivo
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Efficient Gene Targeting Mediated by Adeno-Associated Virus and DNA Double-Strand Breaks
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Adeno‐associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo†‡
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Nonrandom Transduction of Recombinant Adeno-Associated Virus Vectors in Mouse Hepatocytes In Vivo: Cell Cycling Does Not Influence Hepatocyte Transduction
Miao, C. H., Nakai, H., Thompson, A. R., Storm, T. A., Chiu, W. S., Snyder, R. O., & Kay, M. A. (2000) · Journal of Virology · doi:10.1128/jvi.74.8.3793-3803.2000 · Cited by 130 · see citing works
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Genetic modifications of the adeno-associated virus type 2 capsid reduce the affinity and the neutralizing effects of human serum antibodies
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Recent advances in insulin gene therapy for type 1 diabetes
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Adeno-Associated Virus Type 2-Mediated Transduction of Human Monocyte-Derived Dendritic Cells: Implications for Ex Vivo Immunotherapy
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Highly Efficient Targeted Transduction of Undifferentiated Human Hematopoietic Cells by Adenoviral Vectors Displaying Fiber Knobs of Subgroup B
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Viral Transduction of Male Germline Stem Cells Results in Transgene Transmission after Germ Cell Transplantation in Pigs1
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Gene Therapy Using Hematopoietic Stem Cells: Sisyphus Approaches the Crest
Halene, S., & Kohn, D. B. (2000) · Human Gene Therapy · doi:10.1089/10430340050032366 · Cited by 64 · see citing works
Successful Gene Transfer Using Adeno-Associated Virus Vectors into the Kidney: Comparison among Adeno-Associated Virus Serotype 1–5 Vectors in vitro and in vivo
Takeda, S., Takahashi, M., Mizukami, H., Kobayashi, E., Takeuchi, K., Hakamata, Y., Kaneko, T., Yamamoto, H., Ito, C., Ozawa, K., et al. (2004) · Nephron Experimental Nephrology · doi:10.1159/000077378 · Cited by 63 · see citing works
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Scalable production of adeno‐associated virus type 2 vectors via suspension transfection
Park, J. Y., Lim, B., Lee, K., Kim, Y. K., & Jo, E. (2006) · Biotechnology and Bioengineering · doi:10.1002/bit.20776 · Cited by 55 · see citing works
Complete restoration of phenylalanine oxidation in phenylketonuria mouse by a self‐complementary adeno‐associated virus vector
Yagi, H., Ogura, T., Mizukami, H., Urabe, M., Hamada, H., Yoshikawa, H., Ozawa, K., & Kume, A. (2011) · The Journal of Gene Medicine · doi:10.1002/jgm.1543 · Cited by 52 · see citing works
Adeno-Associated Virus Vectors for Therapeutic Gene Transfer
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Kinetics of Recombinant Adeno-Associated Virus-Mediated Gene Transfer
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Authors: Russell, D. W., & Kay, M. A.
Affiliations: University of Washington (US)
ORCID: Kay, Mark A.
Venue: Blood
Publisher: American Society of Hematology
Published: 1999-08-01
Volume: 94
Issue: 3
Pages: 864-874
ISSN: 0006-4971 (ISSN-L)
1528-0020
DOI: 10.1182/blood.v94.3.864.415k34_864_874
PMC: PMC3739711
OpenAlex: W1519039806
Cited by: 104
References: 137
Citations by year: 2026: 1 · 2024: 1 · 2023: 3 · 2021: 1 · 2020: 1 · 2019: 2 · 2018: 1 · 2017: 2 · 2016: 1 · 2015: 1 · 2014: 4 · 2012: 4
FWCI: 6.18
Type: journal article
Language: en
Open access: green
www.ncbi.nlm.nih.gov
Locations: Blood (publishedVersion)
PubMed (publishedVersion)
Europe PMC (PubMed Central) (acceptedVersion)
PubMed Central (acceptedVersion)
Funders: National Heart, Lung, and Blood Institute (P01HL053750, HL 53682, HL53750, R01HL053682)
National Institute of Diabetes and Digestive and Kidney Diseases (R01DK055759)
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
Health Sciences › Medicine › Hematology › Hemophilia Treatment and Research
Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
Keywords: adeno-associated virus vectors, hematopoietic stem cells, gene therapy, retroviral vectors, factor IX, hemophilia B, erythropoietin, hematocrit, secreted proteins, cytokines, coagulation factors
MeSH terms: Dependovirus, Animals, Genetic Vectors, Hematologic Diseases, Humans, Genetic Therapy, Gene Transfer Techniques

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