Gene Editing of the Ube3a Antisense-Transcript in an Angelman Syndrome Mouse Model

被引:0
|
作者
Schmid, Ralf S. [1 ]
Deng, Xuefeng [1 ]
Msackyi, Msema [1 ]
Wilson, James M. [1 ]
机构
[1] Univ Penn, Dept Med, 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
934
引用
收藏
页码:405 / 405
页数:1
相关论文
共 50 条
  • [1] Antisense oligonucleotide treatment rescues UBE3A expression and multiple phenotypes of an Angelman syndrome mouse model
    Milazzo, Claudia
    Mientjes, Edwin J.
    Wallaard, Ilse
    Rasmussen, Soren Vestergaard
    Erichsen, Kamille Dumong
    Kakunuri, Tejaswini
    van der Sman, A. S. Elise
    Kremer, Thomas
    Miller, Meghan T.
    Hoener, Marius C.
    Elgersma, Ype
    JCI INSIGHT, 2021, 6 (15)
  • [2] Angelman Syndrome Due to UBE3A Gene Mutation
    Jyotindra Narayan Goswami
    Jitendra Kumar Sahu
    Pratibha Singhi
    The Indian Journal of Pediatrics, 2018, 85 : 390 - 391
  • [3] Angelman Syndrome Due to UBE3A Gene Mutation
    Goswami, Jyotindra Narayan
    Sahu, Jitendra Kumar
    Singhi, Pratibha
    INDIAN JOURNAL OF PEDIATRICS, 2018, 85 (05): : 390 - 391
  • [4] Generation and Characterization of a Novel Angelman Syndrome Mouse Model with a Full Deletion of the Ube3a Gene
    Syding, Linn Amanda
    Kubik-Zahorodna, Agnieszka
    Nickl, Petr
    Novosadova, Vendula
    Kopkanova, Jana
    Kasparek, Petr
    Prochazka, Jan
    Sedlacek, Radislav
    CELLS, 2022, 11 (18)
  • [5] Gene therapy "unsilences" paternal Ube3a in Angelman syndrome model
    Dengler, V. L.
    GENETICS IN MEDICINE, 2021, 23 (03) : 428 - 428
  • [6] CRISPR/Cas9 directed to the Ube3a antisense transcript improves Angelman syndrome phenotype in mice
    Schmid, Ralf S.
    Deng, Xuefeng
    Panikker, Priyalakshmi
    Msackyi, Msema
    Breton, Camilo
    Wilson, James M.
    JOURNAL OF CLINICAL INVESTIGATION, 2021, 131 (05):
  • [7] SNURF-SNRPN and UBE3A transcript levels in patients with Angelman syndrome
    Runte, M
    Kroisel, PM
    Gillessen-Kaesbach, G
    Varon, R
    Horn, D
    Cohen, MY
    Wagstaff, J
    Horsthemke, B
    Buiting, K
    HUMAN GENETICS, 2004, 114 (06) : 553 - 561
  • [8] Imprinting of the Angelman syndrome gene, UBE3A, is restricted to brain
    Thanh H. Vu
    Andrew R. Hoffman
    Nature Genetics, 1997, 17 : 12 - 13
  • [9] SNURF-SNRPN and UBE3A transcript levels in patients with Angelman syndrome
    Maren Runte
    Peter M. Kroisel
    Gabriele Gillessen-Kaesbach
    Raymonda Varon
    Denise Horn
    Monika Y. Cohen
    Joseph Wagstaff
    Bernhard Horsthemke
    Karin Buiting
    Human Genetics, 2004, 114 : 553 - 561
  • [10] Screening of UBE3A gene in patients referred for Angelman Syndrome
    Tzagkaraki, Evmorfia
    Sofocleous, Christalena
    Helen, Fryssira-Kanioura
    Dinopoulos, Argyris
    Goulielmos, Georgios
    Mavrou, Ariadni
    Sofia, Kitsiou-Tzeli
    Kanavakis, Emmanuel
    EUROPEAN JOURNAL OF PAEDIATRIC NEUROLOGY, 2013, 17 (04) : 366 - 373