Zobrazeno 1 - 10
of 153
pro vyhledávání: '"adeno-associated virus vectors"'
Publikováno v:
Биопрепараты: Профилактика, диагностика, лечение, Vol 24, Iss 2, Pp 123-139 (2024)
INTRODICTION. Currently, gene therapy based on adeno-associated virus (AAV) vectors faces a number of barriers, both biomedical and technological, which require studying and overcoming for further development of this gene therapy technology.AIM. This
Externí odkaz:
https://doaj.org/article/71be6780a74542e89f7c57e1138ef4ad
Autor:
Yuji Kashiwakura, Kazuhiro Endo, Atsushi Ugajin, Tomohiro Kikuchi, Shuji Hishikawa, Hitoyasu Nakamura, Yuko Katakai, Nemekhbayar Baatartsogt, Takafumi Hiramoto, Morisada Hayakawa, Nobuhiko Kamoshita, Shoji Yamazaki, Akihiro Kume, Harushi Mori, Naohiro Sata, Yoichi Sakata, Shin-ichi Muramatsu, Tsukasa Ohmori
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 30, Iss , Pp 502-514 (2023)
Gene therapy using adeno-associated virus (AAV)-based vectors has become a realistic therapeutic option for hemophilia. We examined the potential of a novel engineered liver-tropic AAV3B-based vector, AAV.GT5, for hemophilia B gene therapy. In vitro
Externí odkaz:
https://doaj.org/article/e60d3f3fd02c459c99665b37f69364fb
Autor:
Benjamin Arnson, Hye Ri Kang, Elizabeth D. Brooks, Dorothy Gheorghiu, Ekaterina Ilich, David Courtney, Jeffrey I. Everitt, Bryan R. Cullen, Dwight D. Koeberl
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 29, Iss , Pp 108-119 (2023)
Glycogen storage disease type Ia (GSD Ia) is the inherited deficiency of glucose-6-phosphatase (G6Pase), associated with life-threatening hypoglycemia and long-term complications, including hepatocellular carcinoma formation. Gene replacement therapy
Externí odkaz:
https://doaj.org/article/8282e120c0924cec897cf976a2665255
Autor:
Christina Wagner, Felix F. Fuchsberger, Bernd Innthaler, Robert Pachlinger, Irene Schrenk, Martin Lemmerer, Ruth Birner-Gruenberger
Publikováno v:
International Journal of Molecular Sciences, Vol 25, Iss 2, p 838 (2024)
Mass photometry (MP) is a fast and simple analysis method for the determination of the proportions of subpopulations in an AAV sample. It is label-free and requires minimal sample volumes between 5–10 µL, which makes it a promising candidate over
Externí odkaz:
https://doaj.org/article/e7a74de3ed6943018bb4c9de128e54bb
Autor:
Christina Wagner, Felix F. Fuchsberger, Bernd Innthaler, Martin Lemmerer, Ruth Birner-Gruenberger
Publikováno v:
International Journal of Molecular Sciences, Vol 24, Iss 13, p 11033 (2023)
Adeno-associated viruses (AAV) are one of the most commonly used vehicles in gene therapies for the treatment of rare diseases. During the AAV manufacturing process, particles with little or no genetic material are co-produced alongside the desired A
Externí odkaz:
https://doaj.org/article/5158139c60044a67858f9d31749dc34a
Autor:
Ana Sofia Coroadinha
Publikováno v:
Cells, Vol 12, Iss 5, p 732 (2023)
Gene therapy relies on the delivery of genetic material to the patient’s cells in order to provide a therapeutic treatment. Two of the currently most used and efficient delivery systems are the lentiviral (LV) and adeno-associated virus (AAV) vecto
Externí odkaz:
https://doaj.org/article/d8345103f16d40d8b310fc02b6a836ef
Akademický článek
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Autor:
Christina Wagner, Bernd Innthaler, Martin Lemmerer, Robert Pletzenauer, Ruth Birner-Gruenberger
Publikováno v:
International Journal of Molecular Sciences, Vol 23, Iss 21, p 12715 (2022)
Ion-exchange chromatography coupled to light scattering detectors represents a fast and simple analytical method for the assessment of multiple critical quality attributes (CQA) in one single measurement. The determination of CQAs play a crucial role
Externí odkaz:
https://doaj.org/article/8973569777f241cbb3c006680e2a0bb7
Autor:
Manish Muhuri, Wei Zhan, Yukiko Maeda, Jia Li, Anoushka Lotun, Jennifer Chen, Katelyn Sylvia, Ishani Dasgupta, Motahareh Arjomandnejad, Thomas Nixon, Allison M. Keeler, Sangeetha Manokaran, Ran He, Qin Su, Phillip W. L. Tai, Guangping Gao
Publikováno v:
Frontiers in Immunology, Vol 12 (2021)
Recombinant adeno-associated virus (rAAV) platforms hold promise for in vivo gene therapy but are undermined by the undesirable transduction of antigen presenting cells (APCs), which in turn can trigger host immunity towards rAAV-expressed transgene
Externí odkaz:
https://doaj.org/article/01510ca4e9254713881c8470321ee966
Akademický článek
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