A first-in-human phase 0 clinical study of RNA interference–based spherical nucleic acids in patients with recurrent glioblastoma

Author:

Kumthekar Priya1ORCID,Ko Caroline H.2ORCID,Paunesku Tatjana3ORCID,Dixit Karan1ORCID,Sonabend Adam M.4ORCID,Bloch Orin4,Tate Matthew4,Schwartz Margaret1,Zuckerman Laura1,Lezon Ray1,Lukas Rimas V.1,Jovanovic Borko5,McCortney Kathleen4ORCID,Colman Howard6ORCID,Chen Si7ORCID,Lai Barry7ORCID,Antipova Olga7ORCID,Deng Junjing7ORCID,Li Luxi7ORCID,Tommasini-Ghelfi Serena1ORCID,Hurley Lisa A.1ORCID,Unruh Dusten4ORCID,Sharma Nitya V.8ORCID,Kandpal Manoj9ORCID,Kouri Fotini M.1,Davuluri Ramana V.9,Brat Daniel J.8,Muzzio Miguel10ORCID,Glass Mitchell11ORCID,Vijayakumar Vinod11,Heidel Jeremy12,Giles Francis J.13ORCID,Adams Ann K.14ORCID,James C. David4ORCID,Woloschak Gayle E.3,Horbinski Craig48ORCID,Stegh Alexander H.12ORCID

Affiliation:

1. Ken and Ruth Davee Department of Neurology, The Northwestern Malnati Brain Tumor Institute, Feinberg School of Medicine, The Robert H. Lurie Comprehensive Cancer Center, Northwestern University, Chicago, IL 60611, USA.

2. International Institute for Nanotechnology, Northwestern University, Evanston, IL 60208, USA.

3. Department of Radiation Oncology, Feinberg School of Medicine, Northwestern University, Chicago, IL 60611, USA.

4. Department of Neurological Surgery, Feinberg School of Medicine, Northwestern University, Chicago, IL 60611, USA.

5. Department of Preventive Medicine, Feinberg School of Medicine, Northwestern University, Chicago, IL 60611, USA.

6. Huntsman Cancer Institute and Department of Neurosurgery, University of Utah, Salt Lake City, UT 84112, USA.

7. X-ray Science Division, Advanced Photon Source, Argonne National Laboratory, Argonne, IL 60439, USA.

8. Department of Pathology, Feinberg School of Medicine, Northwestern University, Chicago, IL 60611, USA.

9. Preventive Medicine, Health and Biomedical Informatics, Feinberg School of Medicine, The Robert H. Lurie Comprehensive Cancer Center, Northwestern University, Chicago, IL 60611, USA.

10. Life Sciences Group, IIT Research Institute, Chicago, IL 60616, USA.

11. Broom Street Associates, Wilmington, DE 19806, USA.

12. Informulate LLC, Madison, WI 53704, USA.

13. Developmental Therapeutics Program of the Division of Hematology Oncology, Feinberg School of Medicine, The Robert H. Lurie Comprehensive Cancer Center, Northwestern University, Chicago, IL 60611, USA.

14. Office for Research, Northwestern University, Evanston, IL 60208, USA.

Abstract

RNA interference–based spherical nucleic acid nanoconjugates are a potentially safe and brain-penetrant approach for glioblastoma treatment.

Funder

National Institutes of Health

NIH Clinical Center

U.S. Department of Energy

Deleted Deleted

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

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