Wang, Xiaoyan Klann, Patrick Julian Wiedtke, Ellen Sano, Yumi Fischer, Nico Schiller, Lisa Elfert, Anna Güttsches, Anne-Katrin Weyen, Ute Grimm, Dirk
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Published in
Frontiers in Immunology
High levels of pre-existing antibodies are a major challenge for the application of viral vectors since they can severely limit their efficacy. To identify promising candidates among adeno-associated virus (AAV) based vectors for future gene therapies for the treatment of hereditary neuromuscular disorders (NMDs), we investigated the antibody level...
Wiggins, Kristin B. Winston, Stephen M. Reeves, Isaiah L. Gaevert, Jessica Spence, Yunyu Brimble, Mark A. Livingston, Brandi Morton, Christopher L. Thomas, Paul G. Sant, Andrea J.
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Published in
Journal of Virology
Developing an improved seasonal influenza vaccine remains an ambitious goal of researchers and clinicians alike. With influenza routinely causing severe epidemics with the potential to rise to pandemic levels, it is critical to create an effective, broadly protective, and durable vaccine to improve public health worldwide. As a potential solution, ...
Wiggins, Kristin B. Winston, Stephen M. Reeves, Isaiah L. Gaevert, Jessica Spence, Yunyu Brimble, Mark A. Livingston, Brandi Morton, Christopher L. Thomas, Paul G. Sant, Andrea J.
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Published in
Journal of Virology
Developing an improved seasonal influenza vaccine remains an ambitious goal of researchers and clinicians alike. With influenza routinely causing severe epidemics with the potential to rise to pandemic levels, it is critical to create an effective, broadly protective, and durable vaccine to improve public health worldwide. As a potential solution, ...
van Dijk, Eva B. Ginn, Samantha L. Alexander, Ian E. Graves, Lara E.
Published in
Exploration of Endocrine and Metabolic Diseases
Congenital adrenal hyperplasia due to 21-hydroxylase deficiency leads to high morbidity and mortality, despite the availability of life-saving corticosteroid replacement therapy. Gene therapy represents a promising potential treatment for monogenic disorders such as congenital adrenal hyperplasia, overcoming the limitations of corticosteroid replac...
Witchey, Shannah Haupt, Alexandra Caldwell, Heather K.
Published in
Frontiers in Neuroscience
In rodents, oxytocin (Oxt) contributes to the onset of maternal care by shifting the perception of pups from aversive to attractive. Both Oxt receptor knockout (Oxtr −/−) and forebrain-specific Oxtr knockout (FB/FB) dams abandon their first litters, likely due to a failure of the brain to ‘switch’ to a more maternal state. Whether this behavioral s...
Borges, Beltran Varthaliti, Antonia Schwab, Marisa Clarke, Maria Pivetti, Christopher Gupta, Nalin Cadwell, Cathryn Guibinga, Ghiabe Phillips, Shirley Del Rio, Tony
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Prenatal somatic cell gene therapy (PSCGT) could potentially treat severe, early-onset genetic disorders such as spinal muscular atrophy (SMA) or muscular dystrophy. Given the approval of adeno-associated virus serotype 9 (AAV9) vectors in infants with SMA by the U.S. Food and Drug Administration, we tested the safety and biodistribution of AAV9-GF...
Sutter, Sereina O. Tobler, Kurt Seyffert, Michael Lkharrazi, Anouk Zöllig, Joël Schraner, Elisabeth M. Vogt, Bernd Büning, Hildegard Fraefel, Cornel
Published in
Journal of Virology
Adeno-associated virus (AAV) vectors are among the most frequently used viral vectors for gene therapy. The lack of pathogenicity of the parental virus, the long-term persistence as episomes in non-proliferating cells, and the availability of a variety of AAV serotypes differing in their cellular tropism are advantageous features of this biological...
Yamamoto, Yutaro Fabbri, Camila Okuhara, Daiki Takagi, Rina Kawabata, Yuna Katayama, Takuto Iyori, Mitsuhiro Hasyim, Ammar A. Sakamoto, Akihiko Mizukami, Hiroaki
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Published in
Frontiers in Immunology
Among Plasmodium spp. responsible for human malaria, Plasmodium vivax ranks as the second most prevalent and has the widest geographical range; however, vaccine development has lagged behind that of Plasmodium falciparum, the deadliest Plasmodium species. Recently, we developed a multistage vaccine for P. falciparum based on a heterologous prime-bo...
Symington, Emily Rangarajan, Savita Lester, Will Madan, Bella Pierce, Glenn F Raheja, Priyanka Robinson, Tara M Osmond, Dane Russell, Chris B Vettermann, Christian
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Published in
Haemophilia : the official journal of the World Federation of Hemophilia
Valoctocogene roxaparvovec uses an adeno-associated virus serotype 5 (AAV5) vector to transfer a factor VIII (FVIII) coding sequence to individuals with severe haemophilia A, providing bleeding protection. To assess safety and efficacy of valoctocogene roxaparvovec 5-6 years post-treatment. In a phase 1/2 trial, adult male participants with severe ...
Bijlani, Swati Pang, Ka Ming Bugga, Lakshmi V. Rangasamy, Sampath Narayanan, Vinodh Chatterjee, Saswati
Published in
Frontiers in Genome Editing
Rett syndrome is an acquired progressive neurodevelopmental disorder caused by de novo mutations in the X-linked MECP2 gene which encodes a pleiotropic protein that functions as a global transcriptional regulator and a chromatin modifier. Rett syndrome predominantly affects heterozygous females while affected male hemizygotes rarely survive. Gene t...
