Genome editing for glycogen storage disease type 1A

被引：0
|
作者：
Koeberl, Dwight D. [1 ,2 ]
机构：
[1] Duke Univ, Sch Med, Dept Pediat, Div Med Genet, Durham, NC USA
[2] Duke Univ, Med Ctr, Dept Mol Genet & Microbiol, Durham, NC USA
来源：
MOLECULAR GENETICS AND METABOLISM | 2024年 / 141卷 / 04期
关键词：
D O I：
10.1016/j.ymgme.2024.108160
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
引用
收藏
页数：1
相关论文
共 50 条
  • [41] Correction of glycogen storage disease type 1a in a mouse model by gene therapy
    Zingone, A
    Hiraiwa, H
    Pan, CJ
    Lin, BC
    Chen, HW
    Ward, JM
    Chou, JY
    JOURNAL OF BIOLOGICAL CHEMISTRY, 2000, 275 (02) : 828 - 832
  • [42] Challenges in Screening for Hepatocellular Carcinoma in the Glycogen Storage Disease Type 1a Population
    Bashir, Mustafa R.
    Merkle, Elmar M.
    Kishnani, Priya S.
    INTERNAL MEDICINE, 2012, 51 (13) : 1811 - 1811
  • [43] A CASE OF GLYCOGEN STORAGE DISEASE TYPE 1a MIMICKING FAMILIAL CHYLOMICRONEMIA SYNDROME
    Olgac, A.
    Okur, I
    Biberoglu, G.
    Ezgu, F. S.
    Tumer, L.
    BALKAN JOURNAL OF MEDICAL GENETICS, 2021, 24 (01) : 103 - 105
  • [44] Clinical course and outcome of glycogen-storage disease type 1a and type 1b
    Eminoglu, Fatma Tuba
    Tumer, Leyla
    Okur, Ilyas
    Ezgu, Fatih Suheyl
    Hasanoglu, Alev
    TURK PEDIATRI ARSIVI-TURKISH ARCHIVES OF PEDIATRICS, 2013, 48 (02): : 117 - 122
  • [45] Bezafibrate Enhances AAV Vector-Mediated Genome Editing in Glycogen Storage Disease Type Ia
    Kang, Hye-Ri
    Waskowicz, Lauren
    Seifts, Andrea M.
    Landau, Dustin J.
    Young, Sarah P.
    Koeberl, Dwight D.
    MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2019, 13 : 265 - 273
  • [46] DIABETES MELLITUS AND GLYCOGEN STORAGE DISEASE TYPE 1A IN AN ADULT FEMALE: A CASE STUDY
    Pendyal, Surekha
    Wons, Jonathan
    Austin, Stephanie
    Kishnani, Priya
    MOLECULAR GENETICS AND METABOLISM, 2016, 117 (03) : 277 - 277
  • [47] A Rare Case of Glycogen Storage Disease Type 1a Presenting with Hemophagocytic Lymphohistiocytosis (HLH)
    Saneifard, Hedyeh
    Shamsian, Bibishahin
    Shakiba, Marjan
    Karizi Zarea, Simin
    Sheikhy, Ali
    CASE REPORTS IN PEDIATRICS, 2020, 2020
  • [48] Molecular genetics of glycogen-storage disease type 1a in Chinese patients of Taiwan
    Wong, LJC
    Hwu, WL
    Dai, P
    Chen, TJ
    MOLECULAR GENETICS AND METABOLISM, 2001, 72 (02) : 175 - 180
  • [49] LIVER TRANSPLANT IN GLYCOGEN STORAGE DISEASE TYPE 1A; A CASE THAT DISPUTES CURRENT GUIDELINES
    Bell, Alexandra
    Al-Mukhtar, Ahmed
    GUT, 2021, 70 : A60 - A61
  • [50] 1176C Polymorphism in Japanese patients with glycogen storage disease type 1a
    Okubo, M
    Horinishi, A
    Murase, T
    Hamada, K
    HUMAN GENETICS, 1999, 104 (02) : 193 - 193
12345