【U1132005】利用iPS技术治疗β-地中海贫血关键问题的研究国家自然基金项目

利用iPS技术治疗β-地中海贫血关键问题的研究

项目批准号： U1132005

批准年份： 2011

学科分类： C0607.基因组学

项目负责人：孙筱放

资助类别：联合基金项目

负责人职称：教授

依托单位：广州医科大学

资助金额： 243万元

关键词：基因修复;诱导分化;β-地中海贫血;诱导多能干细胞

起止时间： 2012-01-01到2015-12-31

中英文摘要
结题摘要
结题报告
项目成果
项目参与人

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序号	标题	类型	作者
1	Hepatocyte-specific Hmgb1 Deletion.	期刊论文	Sun X\|Sun X\|Tang D.\|Tang D.\|
2	Hepatocyte-specific Hmgb1 Deletion.	期刊论文	Sun X\|Sun X\|Tang D.\|Tang D.\|
3	Modeling Dravet syndrome using induced pluripotent stem cells (iPSCs) and directly converted neurons.	期刊论文	Yao H\|Liao W\|Sun XF\|Gao S\|
4	High-resolution chromosomal microarray analysis of early-stage human embryonic stem cells reveals an association between X chromosome instability and skewed X inactivation	期刊论文	Li S\|Li S\|Sun X\|Sun X\|
5	Alteration of Scn3a expression is mediated via CpG methylation and MBD2 in mouse hippocampus during postnatal development and seizure condition	期刊论文	Yi YH\|Liao WP\|Sun XF\|Long YS\|
6	无饲养层和动物源蛋白的β-地中海贫血诱导多能干细胞系的建立	期刊论文	范勇\|骆玉梅\|陈欣杰\|孙筱放\|
7	Poly-ADP-ribosylation of HMGB1 regulates TNFSF10/TRAIL resistance through autophagy.	期刊论文	Yang L\|Zhu S\|Cao L\|Tang D\|
8	Genetic Evaluation of Copy Number Variations, Loss of Heterozygosity and Single Nucleotide Variant Levels in Human Embryonic Stem Cells with or without Skewed X Chromosome Inactivation	期刊论文	Wu Bai-Lin\|Wu Bai-Lin\|Sun Xiao-Fang\|Sun Xiao-Fang\|
9	Modeling induced pluripotent stem cells from fibroblasts of Duchenne muscular dystrophy patients.	期刊论文	Chen, Yaoyong\|Chen, Yaoyong\|Sun, Xiaofang\|Sun, Xiaofang\|
10	Diploid, but not haploid, human embryonic stem cells can be derived from microsurgically repaired tripronuclear human zygotes.	期刊论文	Li R\|Huang J\|Yu Y\|Qiao J\|
11	Aberrant expression of maternal PLK1 and Dctn3 results in the developmental failure of human in-vivo- and in-vitro-matured oocytes	期刊论文	Fan Y; Liu JQ;Sun XF\|
12	Direct Conversion of Adipocyte Progenitors into Functional Neurons	期刊论文	Sun, Xiao-Fang\|Sun, Xiao-Fang\|Gao, Shaorong\|Gao, Shaorong\|
13	细胞重编程前后基因组的动态稳定性	期刊论文	范勇\|范勇\|孙筱放\|孙筱放\|
14	细胞重编程前后基因组的动态稳定性	期刊论文	范勇\|范勇\|孙筱放\|孙筱放\|
15	评价羊水染色体检测在产前诊断中的作用	期刊论文	王鼎\|王鼎\|赖炜强\|赖炜强\|
16	High-resolution chromosomal microarray analysis of early-stage human embryonic stem cells reveals an association between X chromosome instability and skewed X inactivation	期刊论文	Li S\|Li S\|Sun X\|Sun X\|
17	Activation of the p62-Keap1-NRF2 pathway protects against ferroptosis in hepatocellular carcinoma cells.	期刊论文	Kang R\|Kang R\|Tang D.\|Tang D.\|
18	丙戊酸钠对SCN3A基因表达的抑制作用	期刊论文	李海军\|李海军\|孙筱放\|孙筱放\|
19	Defining Differentially Methylated Regions Specific for the Acquisition of Pluripotency and Maintenance in Human Pluripotent Stem Cells via Microarray	期刊论文	Wang, Ding\|Sun, Xiaofang\|Yu, Yang\|Fan, Yong\|
20	Prenatal diagnosis of complete maternal uniparental isodisomy of chromosome 4 in a fetus without congenital abnormality or inherited disease-associated variations.	期刊论文	Liu WQ;Zhang HM;Sun XF\|Liu WQ;Zhang HM;Sun XF\|
21	诱导多能干细胞在血液系统疾病中研究进展	期刊论文	范荻;孙筱放\|范荻;孙筱放\|
22	以4种基因诱导产前诊断绒毛细胞建立诱导性多能干细胞	期刊论文	马晓燕\|马晓燕\|孙筱放\|孙筱放\|
23	建立非基因整合iPSC体系及提高体外造血分化体系的研究	期刊论文	范荻;牛晓华;何文茵;孙筱放\|范荻;牛晓华;何文茵;孙筱放\|
24	评价羊水染色体检测在产前诊断中的作用	期刊论文	王鼎\|王鼎\|赖炜强\|赖炜强\|
25	Clinical outcome: a monosomy 18p is better than a tetrasomy 18p.	期刊论文	Wang X\|Wang X\|Sun X\|Sun X\|
26	Prenatal diagnosis of complete maternal uniparental isodisomy of chromosome 4 in a fetus without congenital abnormality or inherited disease-associated variations.	期刊论文	Liu WQ;Zhang HM;Sun XF\|Liu WQ;Zhang HM;Sun XF\|
27	Clinical outcome: a monosomy 18p is better than a tetrasomy 18p.	期刊论文	Wang X\|Wang X\|Sun X\|Sun X\|
28	Improved hematopoietic differentiation efficiency of gene-corrected beta-thalassemia induced pluripotent stem cells by CRISPR/Cas9 system.	期刊论文	Luo M\|Luo M\|Sun X\|Sun X\|
29	植物凝集素刺激外周血单个核细胞增殖及分泌因子表达的变化	期刊论文	孙筱放\|孙筱放\|范勇\|范勇\|
30	Rapid Diagnosis of Imprinting Disorders Involving Copy Number Variation and Uniparental Disomy Using Genome-Wide SNP Microarrays.	期刊论文	Chen M\|Chen M\|Sun X.\|Sun X.\|
31	Generation of induced pluripotent stem cells from human amniotic fluid cells by reprogramming with two factors in feeder-free conditions.	期刊论文	Li Q\|Fan Y\|Sun X\|Yu Y\|
32	应用双胎羊水多能干细胞建立诱导分化的内对照模型	期刊论文	ZHENG Yu-hong\|ZHENG Yu-hong\|SUN Xiao-fang\|SUN Xiao-fang\|
33	GATA2(-/-) human ESCs undergo attenuated endothelial to hematopoietic transition and thereafter granulocyte commitment.	期刊论文	Sun X\|Wang J\|Pei D\|Pan G\|
34	Rapid Diagnosis of Imprinting Disorders Involving Copy Number Variation and Uniparental Disomy Using Genome-Wide SNP Microarrays.	期刊论文	Chen M\|Chen M\|Sun X.\|Sun X.\|
35	Na？ve Induced Pluripotent Stem Cells Generated From β-Thalassemia Fibroblasts Allow Efficient Gene Correction With CRISPR/Cas9.	期刊论文	Sun XF\|Jiang C\|Wang Y\|Gao S\|
36	Genetic Evaluation of Copy Number Variations, Loss of Heterozygosity and Single Nucleotide Variant Levels in Human Embryonic Stem Cells with or without Skewed X Chromosome Inactivation	期刊论文	Wu Bai-Lin\|Wu Bai-Lin\|Sun Xiao-Fang\|Sun Xiao-Fang\|
37	孕中期羊水染色体产前诊断异常检出率与临床诊断相关性的研究	期刊论文	王鼎;孔舒孙筱放\|王鼎;孔舒孙筱放\|
38	Activation of the p62-Keap1-NRF2 pathway protects against ferroptosis in hepatocellular carcinoma cells.	期刊论文	Kang R\|Kang R\|Tang D.\|Tang D.\|
39	Improved hematopoietic differentiation efficiency of gene-corrected beta-thalassemia induced pluripotent stem cells by CRISPR/Cas9 system	期刊论文	Wang D\|Ou Z\|Luo M\|Sun X\|
40	HMGB1-dependent and -independent autophagy	期刊论文	Sun X\|Tang D\|
41	Modification of PCR conditions and design of exon-specific primers for the efficient molecular diagnosis of PKD1 mutations.	期刊论文	Sun X\|Sun X\|Shi Y.\|Shi Y.\|
42	应用双胎羊水多能干细胞建立诱导分化的内对照模型	期刊论文	ZHENG Yu-hong\|ZHENG Yu-hong\|SUN Xiao-fang\|SUN Xiao-fang\|
43	Generation of human β-thalassemia induced pluripotent stem cells from amniotic fluid cells using a single excisable lentiviral stem cell cassette.	期刊论文	Luo Y\|Chen X\|Li Q\|Sun X\|
44	建立非基因整合iPSC体系及提高体外造血分化体系的研究	期刊论文	范荻;牛晓华;何文茵;孙筱放\|范荻;牛晓华;何文茵;孙筱放\|
45	Generation of Induced Pluripotent Stem Cells from Human Amniotic Fluid Cells by Reprogramming with Two Factors in Feeder-free Conditions	期刊论文	Li, Qing\|Fan, Yong\|Sun, Xiaofang\|Yu, Yanhong\|
46	不同培养体系的iPS细胞分化为造血干细胞的差异研究	期刊论文	范荻;何文茵;牛晓华;欧展辉;陈玉嫦;孙筱放\|范荻;何文茵;牛晓华;欧展辉;陈玉嫦;孙筱放\|
47	Improved Efficiency of Microsurgical Enucleated Tripronuclear Zygotes Development and Embryonic Stem Cell Derivation by Supplementing Epidermal Growth Factor, Brain-Derived Neurotrophic Factor, and Insulin-Like Growth Factor-1	期刊论文	Ding, Ting\|Sun, Xiaofang\|Yu, Yang\|Qiao, Jie\|
48	Effects of Integrating and Non-Integrating Reprogramming Methods on Copy Number Variation and Genomic Stability of Human Induced Pluripotent Stem Cells.	期刊论文	Gao X\|He W\|Sun X\|Fan Y\|
49	Improved hematopoietic differentiation efficiency of gene-corrected beta-thalassemia induced pluripotent stem cells by CRISPR/Cas9 system.	期刊论文	Luo M\|Luo M\|Sun X\|Sun X\|
50	孕中期羊水染色体产前诊断异常检出率与临床诊断相关性的研究	期刊论文	王鼎;孔舒孙筱放\|王鼎;孔舒孙筱放\|
51	高龄孕妇脐血遗传产前诊断的成功率及异常率分析	期刊论文	范勇\|范勇\|孙筱放\|孙筱放\|
52	诱导多能干细胞在血液系统疾病中研究进展	期刊论文	范荻;孙筱放\|范荻;孙筱放\|
53	高龄孕妇脐血遗传产前诊断的成功率及异常率分析	期刊论文	范勇\|范勇\|孙筱放\|孙筱放\|
54	多次退火环状循环扩增技术在单细胞水平诊断脊髓性肌萎缩症的应用评估	期刊论文	胡丹;刘维强;张慧敏;刘见桥;杜红姿;梁力丰;孙筱放\|胡丹;刘维强;张慧敏;刘见桥;杜红姿;梁力丰;孙筱放\|
55	丙戊酸钠对SCN3A基因表达的抑制作用	期刊论文	李海军\|李海军\|孙筱放\|孙筱放\|
56	Modeling induced pluripotent stem cells from fibroblasts of Duchenne muscular dystrophy patients	期刊论文	Yu, Bolan\|Li, Qing\|Chen, Yaoyong\|Sun, Xiaofang\|
57	Generation of induced pluripotent stem cells from Asian patients with chronic neurodegenerative diseases.	期刊论文	Chen Y\|Chen Y\|Sun X\|Sun X\|
58	Improved efficiency of microsurgical enucleated tripronuclear zygotes development and embryonic stem cell derivation by supplementing epidermal growth factor, brain-derived neurotrophic factor, and insulin-like growth factor-1	期刊论文	Ding T\|Sun X\|Yu Y\|Qiao J\|
59	Modeling Dravet syndrome using induced pluripotent stem cells (iPSCs) and directly converted neurons.	期刊论文	Gao R\|Fan Y\|Sun X\|Gao S\|
60	不同组织来源的细胞建立诱导多能干细胞的研究	期刊论文	孙筱放\|
61	Generation of Induced Pluripotent Stem Cells from Human Amniotic Fluid Cells by Reprogramming with Two Factors in Feeder-free Conditions	期刊论文	Li Q\| Fan Y\| Sun X\| Yu Y\|
62	Direct Conversion of Adipocyte Progenitors into Functional Neurons	期刊论文	Sun, Xiao-Fang\|Sun, Xiao-Fang\|Gao, Shaorong\|Gao, Shaorong\|
63	Direct conversion of adipocyte progenitors into functional neurons.	期刊论文	Gao R\|Yao H\|孙筱放\|Gao S\|
64	HSPB1 as a Novel Regulator of Ferroptotic Cancer Cell Death	期刊论文	Niu XH\|Wang HC\|Cao LZ\|Tang DL\|
65	Generation of Human beta-thalassemia Induced Pluripotent Stem Cells from Amniotic Fluid Cells Using a Single Excisable Lentiviral Stem Cell Cassette	期刊论文	Luo, Yumei\|Chen, Xinjie\|Li, Qing\|Sun, Xiaofang\|
66	Generation of induced pluripotent stem cells from Asian patients with chronic neurodegenerative diseases.	期刊论文	Chen Y\|Chen Y\|Sun X\|Sun X\|
67	Integrative Analysis of CRISPR/Cas9 Target Sites in the Human HBB Gene.	期刊论文	Chen Y\|Chen Y\|Sun X\|Sun X\|
68	Generation of induced pluripotent stem cells from asian patients with chronic neurodegenerative diseases	期刊论文	孙筱放\|孙筱放\|
69	多次退火环状循环扩增技术在单细胞水平诊断脊髓性肌萎缩症的应用评估	期刊论文	胡丹;刘维强;张慧敏;刘见桥;杜红姿;梁力丰;孙筱放\|胡丹;刘维强;张慧敏;刘见桥;杜红姿;梁力丰;孙筱放\|
70	Integrative Analysis of CRISPR/Cas9 Target Sites in the Human HBB Gene.	期刊论文	Chen Y\|Chen Y\|Sun X\|Sun X\|
71	Modification of PCR conditions and design of exon-specific primers for the efficient molecular diagnosis of PKD1 mutations.	期刊论文	Sun X\|Sun X\|Shi Y.\|Shi Y.\|
72	Modeling induced pluripotent stem cells from fibroblasts of Duchenne muscular dystrophy patients.	期刊论文	Chen, Yaoyong\|Chen, Yaoyong\|Sun, Xiaofang\|Sun, Xiaofang\|
73	不同培养体系的iPS细胞分化为造血干细胞的差异研究	期刊论文	范荻;何文茵;牛晓华;欧展辉;陈玉嫦;孙筱放\|范荻;何文茵;牛晓华;欧展辉;陈玉嫦;孙筱放\|
74	Posttranslational modification of autophagy-related proteins in macroautophagy.	期刊论文	Zhong M\|Huang J\|Klionsky DJ\|Tang D\|
75	Generation of induced pluripotent stem cells from asian patients with chronic neurodegenerative diseases	期刊论文	孙筱放\|孙筱放\|
76	植物凝集素刺激外周血单个核细胞增殖及分泌因子表达的变化	期刊论文	孙筱放\|孙筱放\|范勇\|范勇\|
77	以4种基因诱导产前诊断绒毛细胞建立诱导性多能干细胞	期刊论文	马晓燕\|马晓燕\|孙筱放\|孙筱放\|

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