发育医学电子杂志  2018, Vol. 6 Issue (3): 141-148,192 DOI: 10.3969/j.issn.2095-5340.2018.03.003 |
|
发育基础
专题笔谈
|
|
|
|
|
|
| 基因编辑在镰状细胞贫血症治疗中的研究进展 |
| 肖茹丹 任云晓 张昭军 方向东 |
| 1. 中国科学院北京基因组研究所基因组科学与信息重点实验室，北京 100101；2. 中国科学院大学 生命科学学院，北京100049；3. 中国科学院大学 中丹学院，北京 101408 |
|
| Research progress of gene editing in the treatment for sickle cell anemia |
| XIAO Ru-dan, REN Yun-xiao, ZHANG Zhao-jun, et al |
|
|
|
摘要 镰状细胞贫血症（sickle-cell anemia，SCD）是血红蛋白遗传缺陷疾病，患者的大部分红细胞呈镰 刀 状。 输 血、羟 基 脲（hydroxyurea，HU）及 异 基因 造 血 干 细 胞 移 植（allogeneic hematopoietic stem cell transplantation，allo-HSCT）等 传 统 的 治 疗 方法因无法根治 SCD、供体匮乏、并发症多等原因，限制了临床应用，迫切需要新的治疗方案。近年来，基 因 编 辑 技 术 给 多 种 疾 病 的 治 疗 提 供 了 新的 思 路。 针 对 SCD β- 珠 蛋 白（hemoglobin beta-chain，HBB）的基因突变，可利用基因编辑技术在人造血干祖细胞中改变珠蛋白基因的表达，从而缓解 SCD 症状。本文将从基因编辑治疗策略，基因编辑技术及相关研究进展方面进行综述，为深入研究基于基因编辑的 SCD 治疗方案提供参考
|
|
| 关键词:
镰状细胞贫血症 ')" href="#"> 镰状细胞贫血症
基因编辑
血红蛋白疾病')" href="#"> 血红蛋白疾病
|
|
收稿日期: 2018-05-07
出版日期: 2018-07-30
发布日期: 2018-08-20
期的出版日期: 2018-07-30
|
| 基金资助: 国家自然科学基金（81700097、81700116、81670109、31471115） |
|
通讯作者:
方向东https://baike.baidu.com/item/%E6%96%B9%E5%90%91%E4%B8%9C/4812857
E-mail: fangxd@big.ac.cn
|
| [1] |
GAO Xiao-hui, MAO Jian. Clinical features of non-oliguric hyperkalemia in extremely low birth weight infants[J]. Journal of Developmental Medicine(Electronic Version), 0, (): 152
. |
| [2] |
Society of Neonatologist, Chinese Medical Doctor Association. Consensus recommendations on the prevention and early management of respiratory distress syndrome in preterm infants[J]. Journal of Developmental Medicine(Electronic Version), 2017, 5(3): 129
-131
. |
| [3] |
Professional Committee of Respiratory, Society of Neonatologist, Chinese Medical Doctor Association. Clinical application recommendations for heated humidified high flow nasal cannula[J]. Journal of Developmental Medicine(Electronic Version), 2017, 5(3): 132
-135
. |
| [4] |
YAN Jun, ZHU Xing-wang, SHI Yuan. Application progress of noninvasive ventilate technique for premature infants[J]. Journal of Developmental Medicine(Electronic Version), 2017, 5(3): 136
-140
. |
| [5] |
GU Min-fang, YANG Chuan-zhong. Progress of intrapartum resuscitation for premature infants[J]. Journal of Developmental Medicine(Electronic Version), 2017, 5(3): 141
-145
. |
| [6] |
LIU Shu-hua, SHEN Yue-bo, LIU Cui-qing, MA Li. The efficacy of pulmonary surfactant for pulmonary function in premature tension pneumothorax[J]. Journal of Developmental Medicine(Electronic Version), 2017, 5(3): 146
-151
. |
| [7] |
GAO Xiao-hui, MAO Jian. Clinical features of non-oliguric hyperkalemia in extremely low birth weight infants[J]. Journal of Developmental Medicine(Electronic Version), 2017, 5(3): 152
-158
. |
| [8] |
XIA Yao-fang, YANG Juan , TIAN Bao-li, et al. Value of amplitude-integrated electroencephalography in monitoring acute period of neonatal bilirubin encephalopathy and prognostic assessment[J]. Journal of Developmental Medicine(Electronic Version), 2017, 5(3): 159
-163
. |
| [9] |
WANG Li-rong, SUN Xiao-yan, ZHU Ruo-xin, et al. Epidemiological investigation and analysis of women aged 40-55 years old with osteoporosis in Gansu province[J]. Journal of Developmental Medicine(Electronic Version), 2017, 5(3): 164
-167
. |
| [10] |
CHEN Ru-yue, SHEN Yun-yan, CHEN Qing , et al. Five cases about Henoch-Schönlein purpura complicated with central nervous system injury in children and literatures review[J]. Journal of Developmental Medicine(Electronic Version), 2017, 5(3): 168
-171
. |
|
|
Viewed |
|
|
|
Full text
|
|
|
|
|
Abstract
|
|
|
|
|
Cited |
|
|
|
|
| |
Shared |
|
|
|
|
| |
Discussed |
|
|
|
|
