学术报告：基于CRISPR/Cas9基因编辑技术的肝脏基因治疗

报告人：张庆硕（美国俄勒冈干细胞中心研究员）

报告时间：2019年5月10日下午4:30

报告地点：医学院19号楼810会议室

主办单位：医学院（转化医学研究院）

欢迎广大师生参加！

张庆硕简历

张庆硕，美国俄勒冈干细胞中心研究员，美国俄勒冈微生物学与分子遗传学系（MMG）助理教授，美国俄勒冈微生物学与分子遗传学系博士后，美国冷泉港实验室博士后

主要研究方向：基因治疗；血液疾病实验疗法；癌症预防

主要发表论文：

1. Zhang QS*, Deater M, Phan N, Marcogliese AN, Major A, Guinan, EC, Grompe M. (2019) “Combination therapy with atorvastatin and celecoxib delays tumor formation in a Fanconi anemia mouse model.” Pediatr. Blood Cancer 66:e27460.

2. Zhang QS* (2017) “Stem cell therapy for Fanconi anemia.” In: Advances in Experimental Medicine and Biology. Springer, Boston, MA

3. Zhang QS*, Tang W, Deater M, Phan N, Marcogliese AN, Li H, Al-Dhalimy M, Major A, Olson S, Monnat RJ Jr, Grompe M. (2016) “Metformin improves defective hematopoiesis and delays tumor formation in Fanconi anemia mice.” Blood. 128(24):2774-2784.

4. Zhang QS*, Deater M, Schubert K, Marquez-Loza L, Pelz C, Sinclair DA, Grompe M. (2015) “The Sirt1 activator SRT3025 expands hematopoietic stem and progenitor cells and improves hematopoiesis in Fanconi anemia mice.” Stem Cell Res. 15(1):130-140.

5. Zhang QS*, Benedetti E, Deater M, Schubert K, Major A, Pelz C, Impey S, Marquez-Loza L, Rathbun RK, Kato S, Bagby GC, Grompe M. (2015) “Oxymetholone therapy of Fanconi anemia suppresses osteopontin transcription and induces hematopoietic stem cell cycling.” Stem Cell Reports 4(1):90-102.

6. Zhang QS*, Marquez-Loza L, Sheehan AM, Watanabe-Smith K, Eaton L, Benedetti E, Major A, Schubert K, Deater M, Joseph E, Grompe M. (2014) “Evaluation of resveratrol and N-acetylcysteine for cancer chemoprevention in a Fanconi anemia murine model.” Pediatr. Blood Cancer 61(4):740-2.

7. Figueiredo N, Chora A, Raquel H, Pejanovic N, Pereira P, Hartleben B, Neves-Costa A, Moita C, Pedroso D, Pinto A, Marques S, Faridi H, Costa P, Gozzelino R, Zhao JL, Soares MP, Gama-Carvalho M, Martinez J, Zhang Q, Döring G, Grompe M, Simas JP, Huber TB, Baltimore D, Gupta V, Green DR, Ferreira JA, Moita LF. (2013) “Anthracyclines Induce DNA Damage Response-Mediated Protection against Severe Sepsis.” Immunity 39(5):874-84.

8. Zhang QS*, Watanabe-Smith K, Schubert K, Major A, Sheehan AM, Marquez-Loza L, Newell AE, Benedetti E, Joseph E, Olson S, Grompe M. (2013) “Fancd2 and p21 function independently in maintaining the size of hematopoietic stem and progenitor cell pool in mice.” Stem Cell Res. 11(2):687-92.

9. Ceccaldi R, Parmar K, Mouly E, Delord M, Kim JM, Regairaz M, Pla M, Vasquez N, Zhang QS, Pondarre C, Peffault de Latour R, Gluckman E, Cavazzana-Calvo M, Leblanc T, Larghero J, Grompe M, Socié G, D'Andrea AD, Soulier J. (2012) "Bone marrow failure in Fanconi anemia is triggered by an exacerbated p53/p21 DNA damage response that impairs hematopoietic stem and progenitor cells." Cell Stem Cell 11(1):36-49.

10. Zhang QS*, Marquez-Loza L, Eaton L, Duncan AW, Goldman DC, Anur P, Watanabe-Smith K, Rathbun RK, Fleming WH, Bagby GC, Grompe M. (2010) “Fancd2-/- mice have hematopoietic defects that can be partially corrected by resveratrol.” Blood 116(24): 5140-8.

11. Wang S, Lu B, Girman S, Duan J, McFarland T, Zhang QS, Grompe M, Adamus G, Appukuttan B, Lund R. (2010) “Non-invasive stem cell therapy in a rat model for retinal degeneration and vascular pathology.” PLoS One. 5(2):e9200.

12. Zhang QS*, Eaton L, Snyder ER, Houghtaling S, Mitchell JB, Finegold M, Van Waes C, Grompe M. (2008) “Tempol protects against oxidative damage and delays epithelial tumor onset in Fanconi anemia mice.” Cancer Res. 68(5):1601-8.

13. Chen G, Li W, Zhang QS, Regulski M, Sinha N, Barditch J, Tully T, Krainer AR, Zhang MQ, Dubnau J. (2008) “Identification of synaptic targets of Drosophila Pumilio.” Plos Computational Biol. 4(2):e1000026.

14. Zhang QS, Manche L, Xu RM, Krainer AR. (2006) “hnRNP A1 associates with telomere ends and stimulates telomerase activity.” RNA 12(6):1116-28.

15. Yao YN, Zhang QS, Yan XZ, Zhu G, Wang ED. (2004) “Escherichia coli tRNAArg4(UCU) induces a constrained conformation of the crucial omega-loop of arginyl-tRNA synthetase.” Biochem. Biophys. Res. Commun. 313(1):129-34.

16. Yao YN, Zhang QS, Yan XZ, Zhu G, Wang ED. (2003) “Substrate-induced conformational changes in Escherichia coli arginyl-tRNA synthetase observed by 19F NMR spectroscopy.” FEBS Lett. 547(1-3):197-200.

17. Zhang QS, Wang ED. (1999) “RNA-protein interactions.” Prog. Biochem. Biophys. 26(2):121-5.

18. Zhang QS, Shen L, Wang ED, Wang YL. (1999) “Biosynthesis and characterization of 4-fluorotryptophan-labeled Escherichia coli arginyl-tRNA synthetase.” J. Protein Chem. 18(2):187-92.

19. Zhang QS, Wang ED, Wang YL. (1998) “The role of tryptophan residues in Escherichia coli arginyl-tRNA synthetase.” Biochim. Biophys. Acta 1387(1-2):136-42.