Engineering adeno-associated viral vectors to evade innate immune and inflammatory responses-Reference-Cited by-同舟云学术

Engineering adeno-associated viral vectors to evade innate immune and inflammatory responses

Published:2021-02-10 Issue:580 Volume:13 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Chan Ying Kai¹²³^ORCID,Wang Sean K.²^ORCID,Chu Colin J.⁴^ORCID,Copland David A.⁴^ORCID,Letizia Alexander J.¹²^ORCID,Costa Verdera Helena⁵⁶^ORCID,Chiang Jessica J.¹²³^ORCID,Sethi Meher¹²³^ORCID,Wang May K.³,Neidermyer William J.³,Chan Yingleong¹²^ORCID,Lim Elaine T.¹²,Graveline Amanda R.¹^ORCID,Sanchez Melinda¹^ORCID,Boyd Ryan F.⁷,Vihtelic Thomas S.⁷,Inciong Rolando Gian Carlo O.⁸^ORCID,Slain Jared M.⁸^ORCID,Alphonse Priscilla J.⁵⁶^ORCID,Xue Yunlu²^ORCID,Robinson-McCarthy Lindsey R.¹²^ORCID,Tam Jenny M.¹²^ORCID,Jabbar Maha H.⁹,Sahu Bhubanananda⁹,Adeniran Janelle F.⁹,Muhuri Manish¹⁰¹¹¹²^ORCID,Tai Phillip W. L.¹⁰¹¹¹²^ORCID,Xie Jun¹⁰¹¹¹²,Krause Tyler B.²,Vernet Andyna¹^ORCID,Pezone Matthew¹,Xiao Ru¹³¹⁴,Liu Tina¹²³,Wang Wei⁹,Kaplan Henry J.⁹¹⁵¹⁶^ORCID,Gao Guangping¹⁰¹¹¹²^ORCID,Dick Andrew D.⁴¹⁷,Mingozzi Federico⁵⁶^ORCID,McCall Maureen A.⁹¹⁸^ORCID,Cepko Constance L.²¹⁹²⁰,Church George M.¹²^ORCID

Affiliation:

1. Wyss Institute for Biologically Inspired Engineering, Harvard University, Boston, MA 02115, USA.

2. Department of Genetics, Blavatnik Institute, Harvard Medical School, Boston, MA 02115, USA.

3. Ally Therapeutics, Cambridge, MA 02139, USA.

4. Academic Unit of Ophthalmology, Translational Health Sciences, University of Bristol, Bristol BS8 1TD, UK.

5. Inserm U974, Sorbonne Universite, Paris 75651, France.

6. Inserm S951 and Genethon, Evry 91000, France.

7. Ophthalmology, Charles River Laboratories, Mattawan, MI 49071, USA.

8. Statistics and Data Science, Charles River Laboratories, Mattawan, MI 49071, USA.

9. Department of Ophthalmology and Visual Sciences, University of Louisville, Louisville, KY 40202, USA.

10. Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA 01605, USA.

11. Li Weibo Institute for Rare Diseases Research, University of Massachusetts Medical School, Worcester, MA 01655, USA.

12. Department of Microbiology and Physiological Systems, University of Massachusetts Medical School, Worcester, MA 01655, USA.

13. Grousbeck Gene Therapy Center, Schepens Eye Research Institute and Massachusetts Eye and Ear Infirmary, Boston, MA 02115, USA.

14. Ocular Genomics Institute, Department of Ophthalmology, Harvard Medical School, Boston, MA 02114, USA.

15. Ocular Sciences LLC, St. Louis, MO 63112, USA.

16. Department of Ophthalmology, Saint Louis University School of Medicine, St. Louis, MO 63104, USA.

17. Institute of Ophthalmology and the National Institute for Health Research Biomedical Research Centre, Moorfields Eye Hospital and University College London, London EC1V 9EL, UK.

18. Department of Anatomical Sciences and Neurobiology, University of Louisville, Louisville, KY 40202, USA.

19. Howard Hughes Medical Institute, Harvard Medical School, Boston, MA 02115, USA.

20. Department of Ophthalmology, Harvard Medical School, Boston, MA 02115, USA.

Abstract

Incorporating TLR9-inhibitory sequences in the AAV vector genome inhibits immunogenicity and enhances transgene expression in multiple animal models.

Funder

National Institutes of Health

Howard Hughes Medical Institute

National Eye Research Centre

European Research Council

Wyss Institute for Biologically Inspired Engineering of Harvard University

Underwood Trust