Rosalind Franklin Society Proudly Announces the 2022 Award Recipient for Human Gene Therapy .

Human Gene TherapyVol. 34, No. 13-14 AnnouncementFree AccessRosalind Franklin Society Proudly Announces the 2022 Award Recipient for Human Gene TherapyHildegund ErtlHildegund ErtlThe Wistar Institute, Philadelphia, PA, USASearch for more papers by this authorPublished Online:17 Jul 2023https://doi.org/10.1089/hum.2023.29249.rfs2022AboutSectionsPDF/EPUB Permissions & CitationsPermissionsDownload CitationsTrack CitationsAdd to favorites Back To Publication ShareShare onFacebookTwitterLinked InRedditEmail The Rosalind Franklin Society (RFS), in partnership with Mary Ann Liebert, Inc., publishers, enthusiastically congratulate our distinguished recipient of the 2022 annualRFS Award in Sciencefor this journal, which recognizes the outstanding research and published work of women and underrepresented minority scientists, physicians, and engineers.Zhiquan Xiang, Klaudia Kuranda, William Quinn, Areski Chekaoui, Robert Ambrose, Mohadeseh Hasanpourghai, Mikhail Novikov, Dakota Newman, Christina Cole, Xiangyang Zhou, Federico Mingozzi, and Hildegund C.J. Ertl, “The Effect of Rapamycin and Ibrutinib on Antibody Responses to Adeno-Associated Virus Vector-Mediated Gene Transfer,” Human Gene Therapy 33, no. 11–12 (June 2022): 614–624, http://doi.org/10.1089/hum.2021.258.AbstractAdeno-associated virus (AAV) vector-mediated gene transfer is lessening the impact of monogenetic disorders. Human AAV gene therapy recipients commonly mount immune responses to AAV or the encoded therapeutic protein, which requires transient immunosuppression. Most efforts to date have focused on blunting AAV capsid-specific T cell responses, which have been implicated in elimination of AAV-transduced cells. Here, we explore the use of immunosuppressants, rapamycin given alone or in combination with ibrutinib to inhibit AAV vector- or transgene product-specific antibody responses. Our results show that rapamycin or ibrutinib given alone reduces primary antibody responses against AAV capsid, but the combination of rapamycin and ibrutinib is more effective, blunts recall responses, and reduces numbers of circulating antibody-secreting plasma cells. The drugs fail to lower B cell memory formation or to reduce the inhibitory effects of pre-existing AAV capsid-specific antibodies on transduction efficiency.BiosketchHildegund Ertl came to The Wistar Institute as an associate professor in 1987. A native of Germany, she received her medical degree from the University of Göttingen. While in medical school, she began her scientific training as a student in the Max Planck Institute of Experimental Medicine. After research fellowships at the Australian National University and the University of Minnesota, Ertl joined the faculty of Harvard University before transitioning to Wistar. She became a full professor at Wistar in 1996 and holds professorships at the University of Pennsylvania School of Medicine and The Children's Hospital of Philadelphia.Dr. Ertl's research centers on two areas of investigation: immune responses to gene transfer vehicles that impede long-term successes of gene therapy, and developing vaccines for an array of diseases and conditions (including AIDS, chronic viral infections, COVID-19, and some forms of cancer) not typically considered to be treated using this approach. These vaccines aim to protect against future infections and look to create new therapies for diseases already affecting people.FiguresReferencesRelatedDetails Volume 34Issue 13-14Jul 2023 InformationCopyright 2023, Mary Ann Liebert, Inc., publishersTo cite this article:Hildegund Ertl.Rosalind Franklin Society Proudly Announces the 2022 Award Recipient for Human Gene Therapy.Human Gene Therapy.Jul 2023.587-587.http://doi.org/10.1089/hum.2023.29249.rfs2022Published in Volume: 34 Issue 13-14: July 17, 2023PDF download