References
1. Maurya S, Sarangi P, Jayandharan GR. Safety of
Adeno-associated virus-based vector-mediated gene therapy-impact of
vector dose. Cancer Gene Ther. 2022.
2. Aksenov S, Roberts JC, Mugundu G, Mueller KT, Bhattacharya
I, Tortorici MA. Current and Next Steps Toward Prediction of Human Dose
for Gene Therapy Using Translational Dose-Response Studies. Clin
Pharmacol Ther. 2021;110(5): 1176-1179.
3. Belov A, Schultz K, Forshee R, Tegenge MA. Opportunities and
challenges for applying model-informed drug development approaches to
gene therapies. CPT Pharmacometrics Syst Pharmacol. 2021;10(4):
286-290.
4. West GB, Woodruff WH, Brown JH. Allometric scaling of
metabolic rate from molecules and mitochondria to cells and mammals.Proc Natl Acad Sci U S A. 2002;99 Suppl 1: 2473-2478.
5. Tang F, Wong H, Ng CM. Rational Clinical Dose Selection of
Adeno-Associated Virus-Mediated Gene Therapy Based on Allometric
Principles. Clin Pharmacol Ther. 2021;110(3): 803-807.
6. Dedrick R, Bischoff KB, Zaharko DS. Interspecies correlation
of plasma concentration history of methotrexate (NSC-740). Cancer
Chemother Rep. 1970;54(2): 95-101.
7. Zou P, Yu Y, Zheng N, et al. Applications of human
pharmacokinetic prediction in first-in-human dose estimation. AAPS
J. 2012;14(2): 262-281.
8. Lisowski L, Staber JM, Wright JF, Valentino LA. The
intersection of vector biology, gene therapy, and hemophilia. Res
Pract Thromb Haemost. 2021;5(6): e12586.
9. Zou P. First-in-patient dose prediction for adeno-associated
virus-mediated hemophilia gene therapy using allometric scaling. 2022,
https://www.biorxiv.org/content/10.1101/2022.07.05.498837v1.article-metrics.
10. Ehrhardt A, Kay MA. A new adenoviral helper-dependent
vector results in long-term therapeutic levels of human coagulation
factor IX at low doses in vivo. Blood. 2002;99(11): 3923-3930.
11. Crudele JM, Finn JD, Siner JI, et al. AAV liver expression
of FIX-Padua prevents and eradicates FIX inhibitor without increasing
thrombogenicity in hemophilia B dogs and mice. Blood.2015;125(10): 1553-1561.
12. Terrance Hawk SL, and Timothy Morris. Formulary for
Laboratory Animals : Blackwell Publishing; 2005.
13. Nathwani AC, Reiss UM, Tuddenham EG, et al. Long-term
safety and efficacy of factor IX gene therapy in hemophilia B. N
Engl J Med. 2014;371(21): 1994-2004.
14. Nathwani AC, Tuddenham EG, Rangarajan S, et al.
Adenovirus-associated virus vector-mediated gene transfer in hemophilia
B. N Engl J Med. 2011;365(25): 2357-2365.
15. Lisowski L, Dane AP, Chu K, et al. Selection and evaluation
of clinically relevant AAV variants in a xenograft liver model.Nature. 2014;506(7488): 382-386.
16. Chen N, Sun K, Chemuturi NV, Cho H, Xia CQ. The Perspective
of DMPK on Recombinant Adeno-Associated Virus-Based Gene Therapy: Past
Learning, Current Support, and Future Contribution. AAPS J.2022;24(1): 31.
17. Greig JA, Nordin JML, White JW, et al. Optimized
Adeno-Associated Viral-Mediated Human Factor VIII Gene Therapy in
Cynomolgus Macaques. Hum Gene Ther. 2018.
18. Monahan PE, Negrier C, Tarantino M, Valentino LA, Mingozzi
F. Emerging Immunogenicity and Genotoxicity Considerations of
Adeno-Associated Virus Vector Gene Therapy for Hemophilia. J Clin
Med. 2021;10(11).
19. Manno CS, Pierce GF, Arruda VR, et al. Successful
transduction of liver in hemophilia by AAV-Factor IX and limitations
imposed by the host immune response. Nat Med. 2006;12(3):
342-347.
20. Amit C. Nathwani ET. Preliminary Results of a Phase I/II
Dose Escalation Trial of Gene Therapy for Haemophilia a Using a Novel
Human Factor VIII Variant. Blood. 2018;132(Supplement 1): 489.
21. Pasi KJ, Laffan M, Rangarajan S, et al. Persistence of
haemostatic response following gene therapy with valoctocogene
roxaparvovec in severe haemophilia A. Haemophilia. 2021;27(6):
947-956.
22. Pasi KJ, Rangarajan S, Mitchell N, et al. Multiyear
Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A. N Engl
J Med. 2020;382(1): 29-40.
23. Rangarajan S, Walsh L, Lester W, et al. AAV5-Factor VIII
Gene Transfer in Severe Hemophilia A. N Engl J Med. 2017;377(26):
2519-2530.
24. George LA, Monahan PE, Eyster ME, et al. Multiyear Factor
VIII Expression after AAV Gene Transfer for Hemophilia A. N Engl J
Med. 2021;385(21): 1961-1973.