Development challenges associated with rAAV-based gene therapies-Reference-Cited by-同舟云学术

Development challenges associated with rAAV-based gene therapies

Published:2021 Issue:2 Volume:46 Page:57-68
ISSN:0388-1350
Container-title:The Journal of Toxicological Sciences
language:en
Short-container-title:J. Toxicol. Sci.

Author:

Bolt Michael W.¹,Brady Joseph T.¹,Whiteley Lawrence O.¹,Khan K. Nasir²

Affiliation:

1. Pfizer Inc., Drug Safety Research and Development, Cambridge, MA, USA

2. Pfizer Inc., Drug Safety Research and Development, Groton, CA, USA

Publisher

Japanese Society of Toxicology

Subject

Toxicology

Link

https://www.jstage.jst.go.jp/article/jts/46/2/46_57/_pdf

Reference57 articles.

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2. Audentes press release. (2020): https://www.joshuafrase.org/get-involved/recensus-study.php

3. Bell, P., Wang, L., Lebherz, C., Flieder, D.B., Bove, M.S., Wu, D., Gao, G.P., Wilson, J.M. and Wivel, N.A. (2005): No evidence for tumorigenesis of AAV vectors in a large-scale study in mice. Mol. Ther., 12, 299-306.

4. BioMarin press release. (2020): BioMarin Receives Complete Response Letter (CRL) from FDA for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A. https://investors.biomarin.com/2020-08-19-BioMarin-Receives-Complete-Response-Letter-CRL-from-FDA-for-Valoctocogene-Roxaparvovec-Gene-Therapy-for-Severe-Hemophilia-A

5. Bolt, M.W., Whiteley, L.O., Lynch, J.L., Lauritzen, B., Fernández de Henestrosa, A.R., MacLachlan, T., Ulrich, P., Philip, B.K., Mahalingaiah, P.K., Fuller, C.L. and Compton, D.R. (2020): Nonclinical Studies that Support Viral Vector-Delivered Gene Therapies: An EFPIA Gene Therapy Working Group Perspective. Mol. Ther. Methods Clin. Dev., 19, 89-98.

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