1. Wang D, Tai PWL, Gao G. Adeno-associated virus
vector as a platform for gene therapy delivery.
Nat Rev Drug Discov 2019;18(5):358–378; doi:
10.1038/s41573-019-0012-9
2. Mendell JR, Al-Zaidy SA, Rodino-Klapac LR, et al.
Current clinical applications of in vivo gene
therapy with AAVs. Mol Ther 2021;29(2):464–488;
doi: 10.1016/j.ymthe.2020.12.007
3. Leebeek FWG, Miesbach W. Gene therapy for
hemophilia: A review on clinical benefit, limitations, and remaining issues. Blood 2021;138(11):
923–931; doi: 10.1182/blood.2019003777
4. Jensen TL, Gøtzsche CR, Woldbye DPD. Current
and future prospects for gene therapy for rare
genetic diseases affecting the brain and spinal
cord. Front Mol Neurosci 2021;14:695937; doi:
10.3389/fnmol.2021.695937
5. Bower JJ, Song L, Bastola P, et al. Harnessing the
natural biology of adeno-associated virus to enhance the efficacy of cancer gene therapy. Viruses
2021;13(7); doi: 10.3390/v13071205
6. Scott LJ. Alipogene tiparvovec: A review of its
use in adults with familial lipoprotein lipase deficiency. Drugs 2015;75(2):175–182; doi: 10.1007/
s40265-014-0339-9
7. Waldrop MA, Kolb SJ. Current treatment options
in neurology-SMA therapeutics. Curr Treat Options
Neurol 2019;21(6):25; doi: 10.1007/s11940-019-0568-z
8. Hauck B, Murphy SL, Smith PH, et al. Undetectable
transcription of cap in a clinical AAV vector: Implications for preformed capsid in immune responses. Mol
Ther 2009;17(1):144–152; doi: 10.1038/mt.2008.227
9. Wright JF. Product-related impurities in clinicalgrade recombinant AAV vectors: Characterization
and risk assessment. Biomedicines 2014;2(1):80–
97; doi: 10.3390/biomedicines2010080
10. Urabe M, Ding C, Kotin RM. Insect cells as a
factory to produce adeno-associated virus type 2
vectors. Hum Gene Ther 2002;13(16):1935–1943;
doi: 10.1089/10430340260355347
11. Cle´ment N, Knop DR, Byrne BJ. Large-scale
adeno-associated viral vector production using a
herpesvirus-based system enables manufacturing
for clinical studies. Hum Gene Ther 2009;20(8):
796–806; doi: 10.1089/hum.2009.094
12. Su W, Patrı´cio MI, Duffy MR, et al. Self-attenuating
adenovirus enables production of recombinant
adeno-associated virus for high manufacturing yield
without contamination. Nat Commun 2022;13(1):
1182; doi: 10.1038/s41467-022-28738-2
13. Knezevic I, Stacey G, Petricciani J. WHO Study
Group on cell substrates for production of bio-
logicals, Geneva, Switzerland, 11–12 June 2007.
Biologicals 2008;36(3):203–211; doi: 10.1016/
j.biologicals.2007.11.005
14. Wang Y, Cooper R, Kiladjian A, et al. A digestionfree method for quantification of residual host cell
DNA in rAAV gene therapy products. Mol Ther
Methods Clin Dev 2019;13:526–531; doi: 10.1016/
j.omtm.2019.05.005
15. WHO. Recommendations for the evaluation of
animal cell cultures as substrates for the manufacture of biological medicinal products and for
the characterization of cell banks. Technical Report Series 2013;No 978, Annex 3.
16. Ayuso E, Mingozzi F, Montane J, et al. High AAV
vector purity results in serotype- and tissueindependent enhancement of transduction efficiency. Gene Ther 2010;17(4):503–510; doi:
10.1038/gt.2009.157
17. Andre´ M, Reghin S, Boussard E, et al. Universal
real-time PCR assay for quantitation and size
evaluation of residual cell DNA in human viral
vaccines. Biologicals 2016;44(3):139–149; doi:
10.1016/j.biologicals.2016.03.002
18. Lecomte E, Tournaire B, Cogne´ B, et al. Advanced
characterization of DNA molecules in rAAV vector
preparations by single-stranded virus nextgeneration sequencing. Mol Ther Nucleic Acids
2015;4(10):e260; doi: 10.1038/mtna.2015.32
19. Hindson BJ, Ness KD, Masquelier DA, et al.
High-throughput droplet digital PCR system for
absolute quantitation of DNA copy number. Anal
Chem 2011;83(22):8604–8610; doi: 10.1021/
ac202028g
20. Duewer DL, Kline MC, Romsos EL, et al. Evaluating droplet digital PCR for the quantification of
human genomic DNA: Converting copies per nanoliter to nanograms nuclear DNA per microliter.
Anal Bioanal Chem 2018;410(12):2879–2887; doi:
10.1007/s00216-018-0982-1
21. Graham FL, Smiley J, Russell WC, et al. Characteristics of a human cell line transformed by DNA
from human adenovirus type 5. J Gen Virol 1977;
36(1):59–74; doi: 10.1099/0022-1317-36-1-59
22. Lin YC, Boone M, Meuris L, et al. Genome dynamics
of the human embryonic kidney 293 lineage in response to cell biology manipulations. Nat Commun
2014;5:4767; doi: 10.1038/ncomms5767
23. Sambrook J, Fritsch EF, T. M. Molecular Cloning:
A Laboratory Manual. Cold Spring Harbor Laboratory Press: New York, NY, USA; 1989.
24. He HJ, Stein EV, DeRose P, et al. Limitations of
methods for measuring the concentration of human genomic DNA and oligonucleotide samples.
Biotechniques 2018;64(2):59–68; doi: 10.2144/
btn-2017-0102
25. Kline MC, Duewer DL. Evaluating droplet digital
polymerase chain reaction for the quantification of
human genomic DNA: Lifting the traceability fog.
Anal Chem 2017;89(8):4648–4654; doi: 10.1021/
acs.analchem.7b00240
26. Shmidt AA, Egorova TV. PCR-based analytical
methods for quantification and quality control of
recombinant adeno-associated viral vector preparations. Pharmaceuticals (Basel) 2021;15(1); doi:
10.3390/ph15010023
27. McBride C, Gaupp D, Phinney DG. Quantifying levels
of transplanted murine and human mesenchymal
stem cells in vivo by real-time PCR. Cytotherapy
2003;5(1):7–18; doi: 10.1080/14653240310000038
28. Funakoshi K, Bagheri M, Zhou M, et al. Highly
sensitive and specific Alu-based quantification of
human cells among rodent cells. Sci Rep 2017;
7(1):13202; doi: 10.1038/s41598-017-13402-3
29. Tai PWL, Xie J, Fong K, et al. Adeno-associated
virus genome population sequencing achieves full
vector genome resolution and reveals humanvector chimeras. Mol Ther Methods Clin Dev
2018;9:130–141; doi: 10.1016/j.omtm.2018.02.002
30. McStay B, Grummt I. The epigenetics of rRNA
genes: From molecular to chromosome biology.
Annu Rev Cell Dev Biol 2008;24:131–157; doi:
10.1146/annurev.cellbio.24.110707.175259
31. Malinovskaya EM, Ershova ES, Golimbet VE, et al.
Copy number of human ribosomal genes with
aging: Unchanged mean, but narrowed range and
decreased variance in elderly group. Front Genet
2018;9:306; doi: 10.3389/fgene.2018.00306
32. U.S. Food and Drug Administration. Characterization and qualification of cell substrates and other
biological materials used in the production of viral
vaccines for infectious disease indications. Silver
Spring, MD; 2010. Available from: https://www.
fda.gov/regulatory-information/search-fda-guidancedocuments/characterization-and-qualification-cellsubstrates-and-other-biological-materials-used-production
[Last accessed: May 17, 2019].
33. Brimble MA, Cheng PH, Winston SM, et al. Preventing packaging of translatable P5-associated
DNA contaminants in recombinant AAV vector
preps. Mol Ther Methods Clin Dev 2022;24:280–
291; doi: 10.1016/j.omtm.2022.01.008
Received for publication January 18, 2023;
accepted after revision April 12, 2023.
Published online: April 13, 2023.
...
論文の公開元へ
