Hematopoietic stem cell transplantation for patients with mucopolysaccharidosis type II

被引：2
|
作者：
Kubaski, Francyne [1 ,2 ]
Suzuki, Yasuyuki [3 ]
Yabe, Hiromasa [3 ]
Mason, Robert W. [1 ,2 ]
Xie, Li [2 ]
Onsten, Tor G. H. [4 ,5 ]
Leistner-Segal, Sandra [4 ,5 ]
Giugliani, Roberto [4 ,5 ]
Dung, Vu C. [6 ]
Ngoc, Can T. B. [6 ]
Ngoc, Can T. B. [6 ]
Yamaguchi, Seiji [7 ]
Montano, Adriana M. [8 ]
Orii, Kenji E. [3 ]
Fukao, Toshiyuki [3 ]
Shinataku, Haruo [9 ]
Orii, Tadao [3 ]
Tomatsu, Shunji [1 ,2 ]
机构：
[1] Univ Delaware, Newark, DE USA
[2] Alfred I duPont Children Hosp, Nemours, Wilmington, DE USA
[3] Gifu Univ, Gifu, Japan
[4] HCPA, Porto Alegre, RS, Brazil
[5] Univ Fed Rio Grande do Sul, Porto Alegre, RS, Brazil
[6] Vietnam Natl Childrens Hosp, Hanoi, Vietnam
[7] Shimane Univ, Izumo, Shimane, Japan
[8] St Louis Univ, St Louis, MO 63103 USA
[9] Osaka City Univ, Grad Sch Med, Osaka, Japan
来源：
MOLECULAR GENETICS AND METABOLISM | 2017年 / 120卷 / 1-2期
关键词：
D O I：
10.1016/j.ymgme.2016.11.184
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
175
引用
收藏
页码：S77 / S77
页数：1
相关论文
共 50 条
  • [21] Genotype of mucopolysaccharidosis type II severe form and the efficacy of enzyme replacement therapy or hematopoietic stem cell transplantation on cognitive function
    Tanaka, Akemi
    Hamazaki, Takashi
    Okuyama, Torayuki
    Sakai, Norio
    Kato, Koji
    Suzuki, Yasuyuki
    Yabe, Hiromasa
    Kosuga, Motomichi
    Shinpo, Michiko
    Ishige, Mika
    Kadono, Chiho
    Kudo, Satoshi
    Sawada, Tomo
    Mugishima, Hideo
    Tabuchi, Ken
    Kato, Shunichi
    MOLECULAR GENETICS AND METABOLISM, 2015, 114 (02) : S111 - S112
  • [22] Hematopoietic stem cell gene therapy for mucopolysaccharidosis type IIIC
    Badell-Grau, Rafael
    Pakravesh, Kasra
    Sivakumar, Anusha
    Losay, Pauline
    Duong, Kalvin
    Tran, Christine
    Pithia, Rushil
    Khare, Veenita
    Thai, Kevin
    Cherqui, Stephanie
    MOLECULAR GENETICS AND METABOLISM, 2023, 138 (02) : 13 - 13
  • [23] Hematopoietic Stem Cell Gene Therapy For Mucopolysaccharidosis Type IIIC
    Badell-Grau, Rafael A.
    Rainaldi, Joseph
    Losay, Pauline
    Doung, Kalvin
    Pithia, Rushil
    Pakravesh, Kasra
    Silva, Alexander
    Thai, Eric
    Sharma, Jay
    Paulchakrabarti, Mousumi
    Khare, Veenita
    Cherqui, Stephanie
    MOLECULAR THERAPY, 2022, 30 (04) : 300 - 301
  • [24] Hematopoietic Stem Cell Gene Therapy for Mucopolysaccharidosis Type IIIC
    Badell-Grau, Rafael A.
    Pakravesh, Kasra
    Chen, Rola
    Thai, Eric
    Son, Frankie
    Duong, Kalvin
    Losay, Pauline
    Pithia, Rushil
    Sivakumar, Anusha
    Khare, Veenita
    Corl, Alexis N.
    Tran, Christine
    Esko, Jefferey
    Cherqui, Stephanie
    MOLECULAR THERAPY, 2024, 32 (04) : 420 - 420
  • [25] Hematopoietic Stem Cell Gene Therapy for Mucopolysaccharidosis Type IIIC
    Badell-Grau, Rafael A.
    Sivakumar, Anusha
    Khare, Veenita
    Duong, Kalvin
    Losay, Pauline
    Pakravesh, Kasra
    Tran, Christine
    Pithia, Rushil
    Lengsfield, Mira
    Thai, Kevin
    Sharma, Jay
    Chen, Rola
    Rainaldi, Joseph N.
    Cherqui, Stephanie
    MOLECULAR THERAPY, 2023, 31 (04) : 325 - 326
  • [26] Is hematopoietic stem cell transplantation a therapeutic option for mucolipidosis type II?
    Ammer, Luise Sophie
    Pohl, Sandra
    Breyer, Sandra Rafaela
    Aries, Charlotte
    Denecke, Jonas
    Perez, Anna
    Petzoldt, Martin
    Schrum, Johanna
    Muller, Ingo
    Muschol, Nicole Maria
    MOLECULAR GENETICS AND METABOLISM REPORTS, 2021, 26
  • [27] Obstructive sleep apnea syndrome after hematopoietic stem cell transplantation in children with mucopolysaccharidosis type I
    Moreau, Johan
    Brassier, Anais
    Amaddeo, Alessandro
    Neven, Benedicte
    Caillaud, Catherine
    Chabli, Allel
    Fernandez-Bolanos, Marta
    Olmo, Jorge
    Valayannopoulos, Vassili
    Fauroux, Brigitte
    MOLECULAR GENETICS AND METABOLISM, 2015, 116 (04) : 275 - 280
  • [28] Obstructive sleep apnea syndrome after hematopoietic stem cell transplantation in children with mucopolysaccharidosis type I
    Valayannopoulos, Vassili
    Brassier, Anais
    Ameddeo, Alessandro
    Neven, Benedicte
    Caillaud, Catherine
    Chabli, Allel
    Fernandez-Bolanos, Marta
    Olmo, Jorge
    Moreau, Johan
    Fauroux, Brigitte
    MOLECULAR GENETICS AND METABOLISM, 2016, 117 (02) : S117 - S117
  • [29] A Mortal Complication in a Case with Mucopolysaccharidosis Type I Following Hematopoietic Stem Cell Transplantation: Pulmonary Haemorrhage
    Yazici, Havva
    Canda, Ebru
    Er, Esra
    Malbora, Baris
    Hismi, Burcu Ozturk
    Onay, Huseyin
    Aksoylar, Serap
    Ucar, Sema Kalkan
    Ozkinay, Ferda
    Coker, Mahmut
    IZMIR DR BEHCET UZ COCUK HASTANESI DERGISI, 2021, 11 (02): : 198 - 201
  • [30] An Innovative Combinatorial Hematopoietic Stem Cell Transplantation Strategy Enhances the Therapeutic Potential of HSC-Based Strategies in Mucopolysaccharidosis Type II
    Ferro, Francesca
    Das, Sabyasachi
    Patel, Janki
    Cavalca, Eleonora
    Yu, Hui
    Peviani, Marco
    Biffi, Alessandra
    MOLECULAR THERAPY, 2018, 26 (05) : 260 - 261
12345