Gene Transfer Properties and Structural Modeling of Human Stem Cell-derived AAV
Open accessAbstract
Adeno-associated virus (AAV) vectors are proving to be remarkably successful for in vivo gene delivery. Based upon reports of abundant AAV in the human marrow, we tested CD34(+) hematopoietic stem cells for the presence of natural AAV. Here, we report for the first time, the presence of novel AAV variants in healthy CD34(+) human peripheral blood stem cells. The majority of healthy peripheral blood stem cell donors were found to harbor AAV in their CD34(+) cells. Every AAV isolated from CD34(+) cells mapped to AAV Clade F. Gene transfer vectors derived from these novel AAVs efficiently underwent entry and postentry processing in human cord blood stem cells and supported stable gene transfer into long-term, in vivo engrafting human HSCs significantly better than other serotypes. AAVHSC-transduced human CD34(+) cells engrafted in vivo and gave rise to differentiated transgene-expressing progeny. Importantly, gene-marked CD34(+) stem cells persisted long term in xenograft recipients, indicating transduction of primitive progenitors. Notably, correlation of structure with function permitted identification of potential capsid components important for HSC transduction. Thus, AAVHSCs represent a new class of genetic vectors for the manipulation of HSC genomes.
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AAVHSCs, like AAV9, are classified into Clade F.
“Like AAV9, they are classified into Clade F”
From Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside (2024) · cites this paper as [128]
AAVHSCs are neurotropic AAVs identified from healthy CD34+ human hematopoietic stem cells.
“AAVHSCs are another group of neurotropic AAVs identified from healthy CD34 + human hematopoietic stem cells [128].”
From Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside (2024) · cites this paper as [128]
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Majewska, A., Siejda, K. W., Kowalec, M., Aleksandrowicz, K., Panchuk, Y., Rogala, M. M., Pianka, K., Minich, A., Jarocka, W., Walędziak, M. S., et al. (2026) · Viruses · doi:10.3390/v18101134
- Authors
- Smith, L. J., Ul-Hasan, T., Carvaines, S. K., Van Vliet, K., Yang, E., Wong, K. K., Agbandje-McKenna, M., & Chatterjee, S.
- Affiliations
- City Of Hope National Medical Center (US)
- Corresponding author
- Chatterjee, Saswati
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2014-09
- Volume
- 22
- Issue
- 9
- Pages
- 1625-1634
- ISSN
- 1525-0016 (ISSN-L)
- PMC
- PMC4435483
- OpenAlex
- W2045343535
- Cited by
- 51
- References
- 40
- Citations by year
- 2026: 1 · 2025: 2 · 2024: 2 · 2023: 4 · 2022: 4 · 2021: 10 · 2020: 3 · 2019: 13 · 2018: 4 · 2017: 3 · 2016: 3 · 2015: 2
- FWCI
- 1.75
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- Locations
- Molecular Therapy (publishedVersion, cc-by-nc-nd)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- Funders
- National Cancer Institute
- National Institutes of Health (P30CA033572, R01HL087285, R01GM082946, P01HL51811)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Health Sciences › Medicine › Hematology › Hematopoietic Stem Cell Transplantation
- Keywords
- adeno-associated virus, adeno-associated virus vectors, hematopoietic stem cells, CD34+ cells, gene transfer, stem cell engraftment, cord blood stem cells, gene therapy vectors
- MeSH terms
- Dependovirus, Animals, Cells, Cultured, Hematopoietic Stem Cells, Humans, Male, Models, Biological, Phylogeny, Transduction, Genetic, Mice, SCID, Hematopoietic Stem Cell Transplantation, Antigens, CD34, Capsid Proteins, Mice
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