Zobrazeno 1 - 10
of 780
pro vyhledávání: '"Vectors in gene therapy"'
Publikováno v:
Journal of Medical Evidence, Vol 4, Iss 1, Pp 46-56 (2023)
Gene therapy is the treatment of abnormal or mutated genes present in cells through the addition of healthy genes or replacement/deletion/site-specific modification of faulty genes. Deoxyribonucleic acid, messenger ribonucleic acid (RNA), small inter
Externí odkaz:
https://doaj.org/article/6128f27a4fa94eac8832b61ca6230ec5
Publikováno v:
Frontiers in Molecular Neuroscience, Vol 16 (2023)
Externí odkaz:
https://doaj.org/article/bdd767871dbd4b0a8eb9bdf69f9cc614
Publikováno v:
Frontiers in Molecular Neuroscience. 16
Advanced therapies as defined by the European Medicines Agency (EMA) comprises strategies involving gene therapy, cell therapy, and tissue engineering. Overall, these strategies offer a wide range of possibilities to treat and cure diseases, includin
Autor:
Sophia H.Y. Liao, Suzanne Scott, Erhua Zhu, Loan Hanh Nguyen, Adrian Westhaus, Adrian J. Thrasher, Grober Baltazar, Renina Gale Navarro, Anais K. Amaya, Marti Cabanes-Creus, Leszek Lisowski, Matthieu Drouyer, Laurence O. W. Wilson, Ian E. Alexander, Matthias Hebben
Publikováno v:
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 24, Iss, Pp 88-101 (2022)
Molecular Therapy: Methods & Clinical Development, Vol 24, Iss, Pp 88-101 (2022)
Recent clinical successes have intensified interest in using adeno-associated virus (AAV) vectors for therapeutic gene delivery. The liver is a key clinical target, given its critical physiological functions and involvement in a wide range of genetic
Autor:
Donald L. Swiderski, Lisa A. Beyer, Diane M. Prieskorn, Xiaobo Ma, Jelka Cimerman, Donna M. Martin, Jingying Guo, Jennifer M. Skidmore, Yehoash Raphael, David F. Dolan
Publikováno v:
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 23, Iss, Pp 319-333 (2021)
Molecular Therapy: Methods & Clinical Development, Vol 23, Iss, Pp 319-333 (2021)
Pathogenic variants in GJB2, the gene encoding connexin 26, are the most common cause of autosomal-recessive hereditary deafness. Despite this high prevalence, pathogenic mechanisms leading to GJB2-related deafness are not well understood, and cures
Autor:
Qingzhong Kong, Emily Dexter
Publikováno v:
Expert Rev Neurother
INTRODUCTION: Cellular prion protein (PrP(C)), some of its derivatives (especially PrP N-terminal N1 peptide and shed PrP), and PrP(C)-containing exosomes have strong neuroprotective activities, which have been reviewed in the companion article (Part
Autor:
Stefan Liebau, Udo Maier, Natalia Pashkovskaia, Stefan Kauschke, Peter Loskill, Alexander Kleger, Julia Roosz, Birgit Stierstorfer, Lena Mesch, Johanna Chuchuy, Matthias J. Düchs, Selin Pars, Sebastian Kreuz, Sophia-Marie Hartmann, Kevin Achberger, Virginia Cora, Madalena Cipriano, Stefan Michelfelder, Christian Schön, Thorsten Lamla, Serena Corti
Publikováno v:
Stem Cell Reports
Summary Gene therapies using adeno-associated viruses (AAVs) are among the most promising strategies to treat or even cure hereditary and acquired retinal diseases. However, the development of new efficient AAV vectors is slow and costly, largely bec
Ultra-sensitive AAV capsid detection by immunocapture-based qPCR following factor VIII gene transfer
Autor:
Fan Yang, Wing Yen Wong, Stephen J. Zoog, Hassibullah Akeefe, Elli Koziol, Christian Vettermann, Jennifer Holcomb, Annie Clark, Kevin Hammon, Joshua Henshaw, Nina Mitchell, Kathryn Patton, Krystal Sandza, Benjamin Kim
Publikováno v:
Gene Therapy. 29:94-105
Adeno-associated virus (AAV)-based gene therapy vectors are replication-incompetent and thus pose minimal risk for horizontal transmission or release into the environment. In studies with AAV5-FVIII-SQ (valoctocogene roxaparvovec), an investigational
Autor:
Takashi Kei Kishimoto, Carsten Brunn
Publikováno v:
Emerging Topics in Life Sciences. 5:597-600
In recent months as vaccines against the SARS-CoV-2 virus continue to rollout across the globe, there has been a renewed interest in ways to activate or ignite the immune system. For a vaccine to be effective, it must be immunogenic and specific to p
Autor:
Yizheng Yao, Yu Tian, Jie Wan, Shun Yao, Shanshan Wang, Lan Huang, Yinqiu Wu, Wenli Chen, Farhana Akter, Xiaochun Zhang
Publikováno v:
Journal of Controlled Release. 333:129-138
The presence of the blood-brain barrier (BBB) remains a challenge in the treatment of central nervous system (CNS) diseases, as it hinders the infiltration of many therapeutic drugs into the brain parenchyma. Therefore, developing efficacious pharmac