Zobrazeno 1 - 10
of 320
pro vyhledávání: '"ex vivo gene therapy"'
Akademický článek
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Akademický článek
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Autor:
Toshiki Tsunogai, Toya Ohashi, Yohta Shimada, Takashi Higuchi, Ayaka Kimura, Ayako M. Watabe, Fusao Kato, Hiroyuki Ida, Hiroshi Kobayashi
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 25, Iss , Pp 448-460 (2022)
GM1-gangliosidosis is a progressive neurodegenerative glycosphingolipidosis resulting from a GLB1 gene mutation causing a deficiency of the lysosomal enzyme β-galactosidase, which leads to the abnormal accumulation of GM1 ganglioside in the central
Externí odkaz:
https://doaj.org/article/7324db0761c5449a83b52405a909083c
Autor:
Masayuki Aso, Tokuo T. Yamamoto, Masayuki Kuroda, Jun Wada, Yoshitaka Kubota, Ko Ishikawa, Yoshiro Maezawa, Naoya Teramoto, Ayako Tawada, Sakiyo Asada, Yasuyuki Aoyagi, Mika Kirinashizawa, Akinobu Onitake, Yuta Matsuura, Kunio Yasunaga, Shun-ichi Konno, Katsuaki Nishino, Misato Yamamoto, Junko Miyoshi, Norihiko Kobayashi, Masami Tanio, Takayuki Ikeuchi, Hidetoshi Igari, Nobuyuki Mitsukawa, Hideki Hanaoka, Koutaro Yokote, Yasushi Saito
Publikováno v:
Heliyon, Vol 8, Iss 11, Pp e11271- (2022)
Background: Familial lecithin: cholesterol acyltransferase (LCAT) deficiency (FLD) is a severe inherited disease without effective treatment. Patients with FLD develop severe low HDL, corneal opacity, hemolytic anemia, and renal injury. Objective: We
Externí odkaz:
https://doaj.org/article/d81d0aaee5b947aba70e26e8afc408b1
Autor:
Adele Mucci, Gabriele Antonarelli, Carolina Caserta, Francesco Maria Vittoria, Giacomo Desantis, Riccardo Pagani, Beatrice Greco, Monica Casucci, Giulia Escobar, Laura Passerini, Nico Lachmann, Francesca Sanvito, Matteo Barcella, Ivan Merelli, Luigi Naldini, Bernhard Gentner
Publikováno v:
EMBO Molecular Medicine, Vol 13, Iss 10, Pp 1-18 (2021)
Abstract The immunosuppressive microenvironment surrounding tumor cells represents a key cause of treatment failure. Therefore, immunotherapies aimed at reprogramming the immune system have largely spread in the past years. We employed gene transfer
Externí odkaz:
https://doaj.org/article/4821b3ab509044fab1a533167e0978e3
Autor:
Masayuki Kuroda, Makoto Hori, Yoshiro Maezawa, Yoshitaka Kubota, Nobuyuki Mitsukawa, Yuki Shiko, Yoshihito Ozawa, Yohei Kawasaki, Yasushi Saito, Hideki Hanaoka, Koutaro Yokote
Publikováno v:
Contemporary Clinical Trials Communications, Vol 28, Iss , Pp 100946- (2022)
Backgrounds: Despite the absolute need for life-long treatment of inherited and genetic diseases, there has been little effort to develop such treatments for most of these conditions due to their rarity. Familial lecithin:cholesterol acyltransferase
Externí odkaz:
https://doaj.org/article/0b3eb7af54c9449696aeeda1d7be6bf9
Autor:
Rustem R Islamov, Farid V Bashirov, Mikhail E Sokolov, Andrei A Izmailov, Filip O Fadeev, Vage A Markosyan, Maria A Davleeva, Olga V Zubkova, Maxim M Smarov, Denis Yu Logunov, Boris S Naroditskyi, Ilnur I Salafutdinov, Albert A Rizvanov, Ramil G Turaev
Publikováno v:
Neural Regeneration Research, Vol 16, Iss 2, Pp 357-361 (2021)
We previously demonstrated that gene-modified umbilical cord blood mononuclear cells overexpressing a combination of recombinant neurotrophic factors are a promising therapeutic approach for cell-mediated gene therapy for neurodegenerative diseases,
Externí odkaz:
https://doaj.org/article/78d62d7f6cd547bebfb841d7401a14b7
Autor:
Miho Wada, Yohta Shimada, Sayoko Iizuka, Natsumi Ishii, Hiromi Hiraki, Toshiaki Tachibana, Kazuhiro Maeda, Mitsuru Saito, Shoutaro Arakawa, Takuya Ishimoto, Takayoshi Nakano, Hiroyuki Ida, Toya Ohashi, Hiroshi Kobayashi
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 19, Iss , Pp 261-274 (2020)
Mucopolysaccharidosis type II is a disease caused by organ accumulation of glycosaminoglycans due to iduronate 2-sulfatase deficiency. This study investigated the pathophysiology of the bone complications associated with mucopolysaccharidosis II and
Externí odkaz:
https://doaj.org/article/d2d11fb78c1542e5850cea2f318246be
Autor:
Celine J. Rocca, Joseph N. Rainaldi, Jay Sharma, Yanmeng Shi, Joseph H. Haquang, Jens Luebeck, Prashant Mali, Stephanie Cherqui
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 17, Iss , Pp 1026-1036 (2020)
Friedreich’s ataxia (FRDA) is an autosomal recessive neurodegenerative disorder caused by expansion of GAA repeats in intron 1 of the frataxin (FXN) gene, leading to significant decreased expression of frataxin, a mitochondrial iron-binding protein
Externí odkaz:
https://doaj.org/article/363e1c35035d4f959e428be557dad5a2
Publikováno v:
Bioengineering, Vol 10, Iss 4, p 422 (2023)
Epidermolysis bullosa (EB) includes a group of rare skin diseases characterized by skin fragility with bullous formation in the skin, in response to minor mechanical injury, as well as varying degrees of involvement of the mucous membranes of the int
Externí odkaz:
https://doaj.org/article/7c6fb977c4d44ad19782a19fc7190633