Correction of Hunter Syndrome in the MPSII Mouse Model by AAV2/8-Mediated Gene Delivery

被引：0
|
作者：
Cardone, Monica
Polito, Vinicia Assunta
Pepe, Stefano
Mann, Linda
D'Azzo, Alessandra
Auricchio, Alberto
Ballabio, Andrea
Cosma, Maria Pia
机构：
[1] TIGEM Telethon Inst Genet & Med, Naples, Italy
[2] St Jude Childrens Res Hosp, Memphis, TN 38105 USA
来源：
MOLECULAR THERAPY | 2006年 / 13卷
关键词：
D O I：
10.1016/j.ymthe.2006.08.234
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：
209
引用
收藏
页码：S81 / S81
页数：1
相关论文
共 50 条
  • [41] In vivo assessment of mutations in OTC for dominant-negative effects following rAAV2/8-mediated gene delivery to the mouse liver
    Ginn, S. L.
    Cunningham, S. C.
    Zheng, M.
    Spinoulas, A.
    Carpenter, K. H.
    Alexander, I. E.
    GENE THERAPY, 2009, 16 (06) : 820 - 823
  • [42] AAV-mediated gene correction in a mutant transgenic LacZ mouse
    Dejneka, NS
    Igoucheva, O
    Alexeev, V
    Maguire, AM
    Pierce, EA
    Yoon, K
    Bennett, J
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2003, 44 : U291 - U291
  • [43] In vivo assessment of mutations in OTC for dominant-negative effects following rAAV2/8-mediated gene delivery to the mouse liver
    S L Ginn
    S C Cunningham
    M Zheng
    A Spinoulas
    K H Carpenter
    I E Alexander
    Gene Therapy, 2009, 16 : 820 - 823
  • [44] Assessment of Toxicity and Biodistribution of an AAV2/8 Vector-Mediated Immunomodulatory Gene Therapy in Mice With Pompe Disease
    Han, Sang-oh
    Wang, Gensheng
    Young, Sarah
    Bali, Deeksha
    Benson, Janet
    Koeberl, Dwight
    MOLECULAR THERAPY, 2014, 22 : S19 - S19
  • [45] An AAV2/5 Vector Enhances Safety of Gene Transfer to the Mouse Salivary Gland
    Geguchadze, R. N.
    Machen, L.
    Zourelias, L.
    Gallo, P. H.
    Passineau, M. J.
    JOURNAL OF DENTAL RESEARCH, 2012, 91 (04) : 382 - 386
  • [46] Correction of maternal phenylketonuria syndrome in the Pahenu2 missense mutant mouse by r-AAV mediated gene therapy
    Laipis, PJ
    Charron, CE
    Embury, JE
    Perera, OP
    Porvasnik, SL
    Fields, CR
    Zori, RT
    MOLECULAR THERAPY, 2004, 9 : S334 - S334
  • [47] Characteristics of AAV2 mediated gene transfer in retinas after optic nerve injury
    Nickells, Robert W.
    Schlamp, Cassandra
    Schmitt, Heather M.
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2017, 58 (08)
  • [48] AAV-mediated gene therapy in a mouse model of sialidosis
    van de Vlekkert, Diantha
    Hu, Huimin
    Weesner, Jason A.
    Gomero, Elida M.
    d'Azzo, Alessandra
    MOLECULAR GENETICS AND METABOLISM, 2023, 138 (02) : 129 - 130
  • [49] AAV2/8 to target the liver for correction of hepatic BH4 and brain monoamine neutrotransmitter deficiency in the Pts-ki/ko mouse model
    Korner, G.
    Adamsen, D.
    Viecelli, H. M.
    Thoeny, B.
    HUMAN GENE THERAPY, 2011, 22 (10) : A49 - A49
  • [50] Disease correction by AAV-mediated gene therapy in a new mouse model of mucopolysaccharidosis type IIID
    Roca, Carles
    Motas, Sandra
    Marco, Sara
    Ribera, Albert
    Sanchez, Victor
    Sanchez, Xavier
    Bertolin, Joan
    Leon, Xavier
    Perez, Jennifer
    Garcia, Miguel
    Villacampa, Pilar
    Ruberte, Jesus
    Pujol, Anna
    Haurigot, Virginia
    Bosch, Fatima
    HUMAN MOLECULAR GENETICS, 2017, 26 (08) : 1535 - 1551
12345