Gene Replacement Therapy for Angelman Syndrome

被引:0
|
作者
Percival, Justin [1 ]
Sengupta, Kasturi [1 ]
Le, Long [1 ]
Arhzaouy, Khalid [1 ]
Born, Heather [1 ]
Buza, Elizabeth [1 ]
Dyer, Cecilia [1 ]
Wilson, James M. [1 ]
机构
[1] Univ Penn, Gene Therapy Program, Philadelphia, PA 19104 USA
关键词
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
160
引用
收藏
页码:83 / 83
页数:1
相关论文
共 50 条
  • [1] Optimizing Gene Therapy in the Treatment of Angelman Syndrome
    Nenninger, A. W.
    Willman, M.
    Willman, J.
    Novoa, M.
    Nash, K. R.
    [J]. CELL TRANSPLANTATION, 2021, 30 : 15 - 16
  • [2] shRNA-Mediated Gene Therapy for the Treatment of Angelman Syndrome
    Kang, Hye Ri
    Zaric, Violeta
    Rahim, Aymun
    DeVries, Samantha P.
    Ma, Yuanqing
    Gumusgoz, Emrah
    Minassian, Berge
    Gray, Steven J.
    Butler, Ryan K.
    [J]. MOLECULAR THERAPY, 2022, 30 (04) : 264 - 264
  • [3] Gene Therapy for Angelman Syndrome: Contemporary Approaches and Future Endeavors
    Tsagkaris, Christos
    Papakosta, Vasiliki
    Miranda, Adriana Viola
    Zacharopoulou, Lefkothea
    Danilchenko, Valeriia
    Matiashova, Lolita
    Dhar, Amrit
    [J]. CURRENT GENE THERAPY, 2020, 19 (06) : 359 - 366
  • [4] Adult Gene Therapy for Epilepsy in a Model of Angelman Syndrome: Hope or Hype?
    Huguenard, John R.
    [J]. EPILEPSY CURRENTS, 2023, 23 (05) : 312 - 314
  • [5] Fishing out the Angelman syndrome gene
    Timothy Donlon
    [J]. Nature Medicine, 1997, 3 : 281 - 281
  • [6] Fishing out the Angelman syndrome gene
    Donlon, T
    [J]. NATURE MEDICINE, 1997, 3 (03) : 281 - 281
  • [7] Improving Gene Therapy for Angelman Syndrome with Secreted Human UBE3A
    Austin W. Nenninger
    Matthew Willman
    Jonathan Willman
    Emma Stewart
    Philippe Mesidor
    Michelle Novoa
    Nicole K Morrill
    Luis Alvarez
    Aurélie Joly-Amado
    Melinda M. Peters
    Danielle Gulick
    Kevin R. Nash
    [J]. Neurotherapeutics, 2022, 19 : 1329 - 1339
  • [8] Benefit of Corticosteroid Therapy in Angelman Syndrome
    Forrest, Katharine M. L.
    Young, Helen
    Dale, Russell C.
    Gill, Deepak S.
    [J]. JOURNAL OF CHILD NEUROLOGY, 2009, 24 (08) : 952 - 958
  • [9] Gene therapy "unsilences" paternal Ube3a in Angelman syndrome model
    Dengler, V. L.
    [J]. GENETICS IN MEDICINE, 2021, 23 (03) : 428 - 428
  • [10] Improving Gene Therapy for Angelman Syndrome with Secreted Human UBE3A
    Nenninger, Austin W.
    Willman, Matthew
    Willman, Jonathan
    Stewart, Emma
    Mesidor, Philippe
    Novoa, Michelle
    Morrill, Nicole K.
    Alvarez, Luis
    Joly-Amado, Aurelie
    Peters, Melinda M.
    Gulick, Danielle
    Nash, Kevin R.
    [J]. NEUROTHERAPEUTICS, 2022, 19 (04) : 1329 - 1339