Zobrazeno 1 - 10
of 26
pro vyhledávání: '"Klaudia Kuranda"'
Autor:
Corinne J. Smith, Nikki Ross, Ali Kamal, Kevin Y. Kim, Elizabeth Kropf, Pascal Deschatelets, Cedric Francois, William J. Quinn, Inderpal Singh, Anna Majowicz, Federico Mingozzi, Klaudia Kuranda
Publikováno v:
Frontiers in Immunology, Vol 13 (2022)
AAV gene transfer is a promising treatment for many patients with life-threatening genetic diseases. However, host immune response to the vector poses a significant challenge for the durability and safety of AAV-mediated gene therapy. Here, we charac
Externí odkaz:
https://doaj.org/article/d9b0d374702f4585b36b080c36e4089e
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 13, Iss , Pp 112-120 (2019)
Hematopoietic stem cell transplantation (HSCT) using unrelated cord blood (CB) donors is a suitable approach when an HLA-matched donor is not available. However, one important drawback is the risk of life-threatening viral infections prior to immune
Externí odkaz:
https://doaj.org/article/7812131664564544b7339ffa6960738a
Autor:
Dounia Djeghloul, Klaudia Kuranda, Isabelle Kuzniak, Daniela Barbieri, Irina Naguibneva, Caroline Choisy, Jean-Christophe Bories, Christine Dosquet, Marika Pla, Valérie Vanneaux, Gérard Socié, Françoise Porteu, David Garrick, Michele Goodhardt
Publikováno v:
Stem Cell Reports, Vol 6, Iss 6, Pp 970-984 (2016)
Summary: The capacity of hematopoietic stem cells (HSC) to generate B lymphocytes declines with age, contributing to impaired immune function in the elderly. Here we show that the histone methyltransferase SUV39H1 plays an important role in human B l
Externí odkaz:
https://doaj.org/article/2bfd3069f5604216847aab88774cdd80
Autor:
Nina Mitchell, Wing Yen Wong, Federico Mingozzi, M. Benjamin Hock, Kelly Lau, Romain Hardet, Mingjin Li, Brian Long, Klaudia Kuranda, Stephen J. Zoog, Gregory M. Hayes, Becky Schweighardt, Christian Vettermann, Philippe Veron
Publikováno v:
Molecular Therapy
Molecular Therapy, Cell Press, 2021, 29 (2), pp.597-610. ⟨10.1016/j.ymthe.2020.12.008⟩
Molecular Therapy, 2021, 29 (2), pp.597-610. ⟨10.1016/j.ymthe.2020.12.008⟩
Molecular Therapy, Cell Press, 2021, 29 (2), pp.597-610. ⟨10.1016/j.ymthe.2020.12.008⟩
Molecular Therapy, 2021, 29 (2), pp.597-610. ⟨10.1016/j.ymthe.2020.12.008⟩
Evaluation of immune responses to adeno-associated virus (AAV)-mediated gene therapies prior to and following dose administration plays a key role in determining therapeutic safety and efficacy. This report describes up to 3 years of immunogenicity d
Autor:
Zhiquan Xiang, Klaudia Kuranda, William Quinn, Areski Chekaoui, Robert Ambrose, Mohadeseh Hasanpourghai, Mikhail Novikov, Dakota Newman, Christina Cole, Xiangyang Zhou, Federico Mingozzi, Hildegund C.J. Ertl
Publikováno v:
Human gene therapy. 33(11-12)
Adeno-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 immunos
Autor:
Yan Li, Hildegund C.J. Ertl, Xiangyang Zhou, Katherine A. High, Klaudia Kuranda, Zhiquan Xiang, Raj K. Kurupati, Federico Mingozzi
Publikováno v:
Molecular Therapy. 28:771-783
Transfer of genes by adeno-associated virus (AAV) vectors is benefiting patients with particular genetic defects. Challenges remain by rejection of AAV-transduced cells, which may be caused by CD8+ T lymphocytes directed to AAV capsid antigens. Reduc
Autor:
Xavier M Anguela, Kristen Jaworski, Stacy E Croteau, John E J Rasko, Tiffany Chang, Federico Mingozzi, Paul E Monahan, Katherine A. High, Kathleen Z Reape, Margaret V Ragni, Lindsey A. George, Amy Macdougall, Spencer K. Sullivan, M Elaine Eyster, Benjamin J. Samelson-Jones, Robert Noble, Michael Recht, Marla Curran, Klaudia Kuranda
Publikováno v:
N Engl J Med
Background The goal of gene therapy for patients with hemophilia A is to safely impart long-term stable factor VIII expression that predictably ameliorates bleeding with the use of the lowest possible vector dose. Methods In this phase 1-2 trial, we
Autor:
Priscilla Jean-Alphonse, Romain Hardet, Philippe Veron, Fanny Collaud, Helena Costa Verdera, Klaudia Kuranda, Federico Mingozzi, Solenne Marmier, Christian Leborgne, Giuseppe Ronzitti
Publikováno v:
Journal of Clinical Investigation
Journal of Clinical Investigation, American Society for Clinical Investigation, 2018, 128 (12), pp.5267-5279. ⟨10.1172/JCI122372⟩
Journal of Clinical Investigation, 2018, 128 (12), pp.5267-5279. ⟨10.1172/JCI122372⟩
Journal of Clinical Investigation, American Society for Clinical Investigation, 2018, 128 (12), pp.5267-5279. ⟨10.1172/JCI122372⟩
Journal of Clinical Investigation, 2018, 128 (12), pp.5267-5279. ⟨10.1172/JCI122372⟩
International audience; Recombinant adeno-associated virus (AAV) vectors have been broadly adopted as a gene delivery tool in clinical trials, owing to their high efficiency of transduction of several host tissues and their low immunogenicity. Howeve
Autor:
Margareth C. Ozelo, Katie Wachtel, Yifeng Chen, Benjamin J. Samelson-Jones, Leslie Raffini, Katherine A. High, Margaret V. Ragni, John E.J. Rasko, Xavier M. Anguela, Lindsey A. George, Jennifer Wellman, Alexa R. Runowski, Federico Mingozzi, Maria Hazbon, Klaudia Kuranda
Publikováno v:
Mol Ther
Adeno-associated virus (AAV) vectors are a leading platform for gene-based therapies for both monogenic and complex acquired disorders. The success of AAV gene transfer highlights the need to answer outstanding clinical questions of safety, durabilit
Publikováno v:
Mol Ther
Molecular Therapy
Molecular Therapy, Cell Press, 2020, ⟨10.1016/j.ymthe.2019.12.010⟩
Molecular Therapy, 2020, ⟨10.1016/j.ymthe.2019.12.010⟩
Molecular Therapy
Molecular Therapy, Cell Press, 2020, ⟨10.1016/j.ymthe.2019.12.010⟩
Molecular Therapy, 2020, ⟨10.1016/j.ymthe.2019.12.010⟩
International audience; Gene therapy with adeno-associated virus (AAV) vectors has demonstrated safety and long-term efficacy in a number of trials across target organs, including eye, liver, skeletal muscle, and the central nervous system. Since the
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::9e2791ab41f4882261c1ed9bb65ae2e4
https://europepmc.org/articles/PMC7054726/
https://europepmc.org/articles/PMC7054726/