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Enzyme replacement in the CSF to treat metachromatic leukodystrophy in mouse model using single intracerebroventricular injection of self-complementary AAV1 vector.
Hironaka, Kohei; Yamazaki, Yoshiyuki; Hirai, Yukihiko; Yamamoto, Motoko; Miyake, Noriko; Miyake, Koichi; Okada, Takashi; Morita, Akio; Shimada, Takashi.
Affiliation
  • Hironaka K; 1] Department of Biochemistry and Molecular Biology; Division of Gene Therapy, Research Center for Advanced Medical Technology; Nippon Medical School, Tokyo, 113-8603, Japan [2] Department of Neurological Surgery; Nippon Medical School, Tokyo, 113-8603, Japan.
  • Yamazaki Y; Department of Biochemistry and Molecular Biology; Division of Gene Therapy, Research Center for Advanced Medical Technology; Nippon Medical School, Tokyo, 113-8603, Japan.
  • Hirai Y; Department of Biochemistry and Molecular Biology; Division of Gene Therapy, Research Center for Advanced Medical Technology; Nippon Medical School, Tokyo, 113-8603, Japan.
  • Yamamoto M; Department of Biochemistry and Molecular Biology; Division of Gene Therapy, Research Center for Advanced Medical Technology; Nippon Medical School, Tokyo, 113-8603, Japan.
  • Miyake N; Department of Biochemistry and Molecular Biology; Division of Gene Therapy, Research Center for Advanced Medical Technology; Nippon Medical School, Tokyo, 113-8603, Japan.
  • Miyake K; Department of Biochemistry and Molecular Biology; Division of Gene Therapy, Research Center for Advanced Medical Technology; Nippon Medical School, Tokyo, 113-8603, Japan.
  • Okada T; Department of Biochemistry and Molecular Biology; Division of Gene Therapy, Research Center for Advanced Medical Technology; Nippon Medical School, Tokyo, 113-8603, Japan.
  • Morita A; Department of Neurological Surgery; Nippon Medical School, Tokyo, 113-8603, Japan.
  • Shimada T; Department of Biochemistry and Molecular Biology; Division of Gene Therapy, Research Center for Advanced Medical Technology; Nippon Medical School, Tokyo, 113-8603, Japan.
Sci Rep ; 5: 13104, 2015 Aug 18.
Article in En | MEDLINE | ID: mdl-26283284
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Genetic Therapy / Cerebroside-Sulfatase / Cerebrospinal Fluid / Adenoviridae / Leukodystrophy, Metachromatic Limits: Animals Language: En Journal: Sci Rep Year: 2015 Type: Article Affiliation country: Japan
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Genetic Therapy / Cerebroside-Sulfatase / Cerebrospinal Fluid / Adenoviridae / Leukodystrophy, Metachromatic Limits: Animals Language: En Journal: Sci Rep Year: 2015 Type: Article Affiliation country: Japan