Optimizing Therapeutic Base Editing for Hereditary Tyrosinemia Type 1

被引：0
|
作者：
Dumitru, Ana Maria [1 ]
Berkowitz, Cara [1 ]
Whittaker, Madelynn [2 ]
Wang, Xiao [3 ]
Kus, Nicole [1 ]
Luks, Valerie [1 ]
Riley, John [1 ]
Carpenter, Marco [1 ]
Archer, Nicholas F. [4 ]
Brooks, Dominique [3 ]
Johnston, William [1 ]
Alameh, Mohamad Gabriel [5 ]
Musunuru, Kiran [2 ]
Peranteau, William [1 ]
机构：
[1] Childrens Hosp Philadelphia, Philadelphia, PA USA
[2] Univ Penn, Philadelphia, PA USA
[3] Univ Penn, Cardiovasc Inst, Perelman Sch Med, Philadelphia, PA USA
[4] Drexel Univ, Coll Med, Philadelphia, PA USA
[5] Univ Penn, Engn mRNA & Targeted Nanomed Core, Philadelphia, PA USA
来源：
MOLECULAR THERAPY | 2023年 / 31卷 / 04期
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：
153
引用
收藏
页码：83 / 84
页数：2
相关论文
共 50 条
  • [41] THE ENZYME DEFECTS IN HEREDITARY TYROSINEMIA TYPE-I
    FURUKAWA, N
    HAYANO, T
    SATO, N
    INOUE, F
    MACHIDA, Y
    KINUGASA, A
    IMASHUKU, S
    KUSUNOKI, T
    TAKAMATISU, T
    JOURNAL OF INHERITED METABOLIC DISEASE, 1984, 7 : 137 - 138
  • [42] Impact of nitisinone treatment and discontinuation on the metabolome of wild type and hereditary tyrosinemia type 1 mice
    Colemonts-Vroninks, Haaike
    Norman, Brendan P.
    Andrew, Davison
    Neuckermans, Jessie
    Lionel, Marcelis
    Anneleen, Mortier
    Dimitri, De Bundel
    Ranganath, Lakshminarayan
    Geert, Martens
    Vanhaecke, Tamara
    James, Gallagher
    De Kock, Joery
    JOURNAL OF HEPATOLOGY, 2020, 73 : S540 - S541
  • [43] CHROMOSOMAL INSTABILITY IN HEREDITARY TYROSINEMIA TYPE-I
    WILSON, KS
    TIMMONS, CF
    HILTON, DS
    WEINBERG, AG
    TONK, V
    PEDIATRIC PATHOLOGY, 1994, 14 (06): : 1055 - 1057
  • [44] NEONATAL SCREEN FOR HEREDITARY TYROSINEMIA TYPE-I
    HOLME, E
    LINDSTEDT, S
    LANCET, 1992, 340 (8823): : 850 - 850
  • [45] Towards therapeutic base editing
    Huiyun Seo
    Jin-Soo Kim
    Nature Medicine, 2018, 24 : 1493 - 1495
  • [46] Towards therapeutic base editing
    Seo, Huiyun
    Kim, Jin-Soo
    NATURE MEDICINE, 2018, 24 (10) : 1493 - 1495
  • [47] LABORATORY MONITORING OF PATIENTS WITH HEREDITARY TYROSINEMIA TYPE I
    Schultz, Matthew
    Netzel, Brian
    Singh, Rani
    Gavrilov, Dimitar
    Oglesbee, Devin
    Raymond, Kimiyo
    Rinaldo, Piero
    Tortorelli, Silvia
    Matern, Dietrich
    MOLECULAR GENETICS AND METABOLISM, 2019, 126 (03) : 324 - 325
  • [48] VISCERAL PATHOLOGY OF HEREDITARY TYROSINEMIA TYPE-I
    RUSSO, P
    OREGAN, S
    AMERICAN JOURNAL OF HUMAN GENETICS, 1990, 47 (02) : 317 - 324
  • [49] LABORATORY MONITORING OF PATIENTS WITH HEREDITARY TYROSINEMIA TYPE I
    Schultz, Matthew
    Netzel, Brian
    Singh, Rani
    Gavrilov, Dimitar
    Oglesbee, Devin
    Raymond, Kimiyo
    Rinaldo, Piero
    Tortorelli, Silvia
    Matern, Dietrich
    MOLECULAR GENETICS AND METABOLISM, 2019, 127 (03) : 298 - 299
  • [50] Laboratory monitoring of patients with hereditary tyrosinemia type I
    Schultz, Matthew J.
    Netzel, Brian C.
    Singh, Rani H.
    Pino, Gisele B.
    Gavrilov, Dimitar K.
    Oglesbee, Devin
    Raymond, Kimiyo M.
    Rinaldo, Piero
    Tortorelli, Silvia
    Smith, Wendy E.
    Matern, Dietrich
    MOLECULAR GENETICS AND METABOLISM, 2020, 130 (04) : 247 - 254
12345