Gradual improvements in the motor and cognitive function after gene therapy for patients with AADC deficiency

被引:0
|
作者
Kojima, K. [1 ]
Miyauchi, A. [1 ]
Nakajima, T. [1 ]
Taga, N. [1 ]
Ono, S. A. [1 ]
Mizukami, H. [1 ]
Kato, M. [3 ]
Osaka, H. [1 ]
Muramatsu, S. [1 ,2 ]
Yamagata, T. [1 ]
机构
[1] Jichi Med Univ, Shimotsuke, Tochigi, Japan
[2] Univ Tokyo, Tokyo, Japan
[3] Showa Univ, Tokyo, Japan
关键词
plasmid DNA; linear polyethylenimine; intratracheal lung delivery; near infrared imaging; bioluminescence imaging; breast cancer;
D O I
暂无
中图分类号
Q81 [生物工程学(生物技术)]; Q93 [微生物学];
学科分类号
071005 ; 0836 ; 090102 ; 100705 ;
摘要
OR06
引用
下载
收藏
页码:A4 / A4
页数:1
相关论文
共 50 条
  • [1] Improvement of Motor and Cognitive Function by Gene Therapy for Patients with AADC Deficiency
    Kojima, Karin
    Yamagata, Takanori
    Muramatsu, Shin-ichi
    Osaka, Hitoshi
    Nakajima, Takeshi
    Taga, Naoyuki
    MOLECULAR THERAPY, 2018, 26 (05) : 399 - 400
  • [2] Improvements in motor function and PET findings following gene transfer to the patients with AADC deficiency
    Yamagata, T.
    Kojima, K.
    Miyauchi, A.
    Nakajima, T.
    Ono, S. A.
    Mizukami, H.
    Ozawa, K.
    Sato, T.
    Kato, M.
    Osaka, H.
    Muramatsu, S.
    HUMAN GENE THERAPY, 2016, 27 (11) : A45 - A45
  • [3] Continuous improvement of motor, autonomic, and cognitive functions following gene therapy for AADC deficiency
    Kojima, K.
    Miyauchi, A.
    Nakajima, T.
    Ono, S. A.
    Mizukami, H.
    Kato, M.
    Osaka, H.
    Muramatsu, S.
    Yamagata, T.
    HUMAN GENE THERAPY, 2016, 27 (11) : A146 - A147
  • [4] AGIL-AADC Gene Therapy Results in Sustained Improvements in Motor and Developmental Milestones Over 5 years in Children With AADC Deficiency
    Chien, Yin-Hsiu
    Lee, Ni-Chung
    Tseng, Sheng-Hong
    Tai, Chun-Hwei
    Conway, Anne Marie
    Pykett, Mark
    Hwu, Wuh-Liang
    NEUROLOGY, 2019, 92 (15)
  • [5] AGIL-AADC gene therapy results in sustained improvements in motor and developmental milestones through 5 years in children with AADC deficiency
    Chien, Y. H.
    Lee, N. C.
    Tseng, S. H.
    Tai, C. H.
    Conway, A. M.
    Pykett, M.
    Hwu, W. L.
    JOURNAL OF THE NEUROLOGICAL SCIENCES, 2019, 405
  • [6] AGIL-AADC Gene Therapy Results in Sustained Improvements in Motor and Developmental Milestones through 5 Years in Children with AADC Deficiency
    Chien, Y-H
    Lee, N-C
    Tseng, S-H
    Tai, C-H
    Conway, A.
    Pykett, M.
    Hwu, W-L
    ANNALS OF NEUROLOGY, 2019, 86 : S136 - S137
  • [7] Gene Therapy for AADC Deficiency
    Pearson, T.
    Gupta, N.
    Viehoever, A.
    Grijalvo-Perez, A.
    Imamura-Ching, J.
    Seo, Y.
    San Sebastian, W.
    Bankiewicz, K.
    ANNALS OF NEUROLOGY, 2018, 84 : S308 - S308
  • [8] Midbrain Gene Therapy for AADC Deficiency
    Bankiewicz, Krystof
    Pearson, Toni
    Kohutnicka, Malgorzata
    Sebastian, Waldy San
    Steel, Dora
    Spaull, Robert
    Soo, Audrey
    Heales, Simon
    Pons, Roser
    Garcia-Cazorla, Angeles
    Ibanez-Mico, Salvador
    Sykut-Cegielska, Jolanta
    Szymanska, Krystyna
    Opladen, Thomas
    Jeltsch, Kathrin
    Oppeboen, Mari
    Testard, Herve
    Roscher, Anne
    Siegert, Sandy
    Willemsen, Michel
    Andrews, Saadet
    Ben-Zeev, Bruria
    Kurian, Manju
    Zabek, Miroslaw
    MOLECULAR THERAPY, 2023, 31 (04) : 4 - 4
  • [9] Gene therapy for children with AADC deficiency
    Wiznitzer, Max
    LANCET CHILD & ADOLESCENT HEALTH, 2017, 1 (04): : 250 - 251
  • [10] Gene therapy takes the fear out of severe AADC deficiency
    Müller T.
    MMW - Fortschritte der Medizin, 2024, 166 (Suppl 1) : 21 - 22