日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

Single nuclei and spatial profiling of sacrococcygeal teratomas reveals cellular composition and X inactivation heterogeneity.

骶尾部畸胎瘤的单核和空间分析揭示了细胞组成和 X 染色体失活的异质性。

Rojas Ernesto J, Giannikou Krinio, Huang Benjamin J, Cho Soo-Jin, Cordero Marco A, Pena Deion, Vu Lan, Bagrodia Aditya, Derderian S Christopher, MacKenzie Tippi C, Laird Diana J

Combinatorial base editing couples disease correction with lineage amplification in hematopoietic stem and progenitor cells

组合碱基编辑技术将疾病矫正与造血干细胞和祖细胞的谱系扩增相结合

Jia, Kun; Soupene, Eric; Sinha, Roshani; Lesch, Benjamin J; Pendergast, Michael A; Choi, Rachel; Zhang, Xinyi; Foppiani, Elisabetta M; Kostamo, Zachary; Chu, Simon N; Sharma, Devesh; Yu, Xinjie; Cordero, Marco; Walters, Mark C; MacKenzie, Tippi C; Sheehan, Vivien A; Cromer, M Kyle

GFP-on mouse model for interrogation of in vivo gene editing.

用于研究体内基因编辑的 GFP 标记小鼠模型

Dib Carla, Queenan Jack A, Swartzrock Leah, Willner Hana, Denis Morgane, Ahmed Nouraiz, Moulana Zada Fareha, Borges Beltran, Charlesworth Carsten T, Lum Tony, Yates Bradley P, Kwon Caleb Y, Scorzo Augustino V, Davis Scott C, Davis Jessie R, He Ran, Xie Jun, Gao Guangping, MacKenzie Tippi C, Liu David R, Newby Gregory A, Czechowicz Agnieszka D

Intra-amniotic antisense oligonucleotide treatment improves phenotypes in preclinical models of spinal muscular atrophy

羊膜腔内反义寡核苷酸治疗可改善脊髓性肌萎缩症临床前模型的表型

Borges, Beltran; Brown, Stephen M; Chen, Wan-Jin; Clarke, Maria T; Herzeg, Akos; Park, Jae Hong; Ross, Joshua; Kong, Lingling; Denton, Madeline; Smith, Amy K; Lum, Tony; Zada, Fareha Moulana; Cordero, Marco; Gupta, Nalin; Cook, Sarah E; Murray, Heather; Matson, John; Klein, Stephanie; Bennett, C Frank; Krainer, Adrian R; MacKenzie, Tippi C; Sumner, Charlotte J

In utero genetic therapy: Treatment of early onset neurological disorders before they start

子宫内基因治疗:在神经系统疾病早期发作前进行治疗

Borges, Beltran; Brown, Stephen M; MacKenzie, Tippi C; Sumner, Charlotte J

Prenatal Delivery of Enzyme Replacement Therapy to Fetuses Affected by Early-Onset Lysosomal Storage Diseases

对患有早发性溶酶体贮积症的胎儿进行产前酶替代疗法

Borges, Beltran; Canepa, Emma; Chang, Irene J; Herzeg, Akos; Lianoglou, Billie; Kishnani, Priya S; Harmatz, Paul; MacKenzie, Tippi C; Cohen, Jennifer L

Evidence for Maternal Autoantibodies in the Pathogenesis of Preterm Birth

母体自身抗体在早产发病机制中的证据

Rackaityte, Elze; Borges, Beltran; Kortbawi, Hannah M; Miller, Haleigh S; Shao, Shirley J; Zorn, Kelsey C; Caspar, Colette; Wong, Rebecca; McCarthy, Frank; Creery, Joseph S; Mayer, Margareta; Kouame, Elaine; Puccinelli, Robert R; Kistler, Amy L; Yu, David J L; Ganem, Sammer Roach; DeRisi, Alexandra; Kawaja, Quinn; Wang, Chung-Yu; Saxena, Aditi; Oltman, Scott; Kung, Andrew F; Mann, Sabrina A; Mitchell, Anthea; Huang, David; Rizzuto, Gabrielle; Baer, Rebecca J; Mandel-Brehm, Caleigh; Ryckman, Kelli K; Gaw, Stephanie L; Hillman, Sara L; Jelliffe-Pawlowski, Laura L; Halkias, Joanna; Sobhani, Nasim C; Anderson, Mark S; Trespidi, Francsesca; Alpin, Claudia; Bosoni, Tiziana; Albertini, Riccardo; Bellingeri, Camilla; Gardella, Barbara; Borghesi, Alessandro; Prince, Nicole; Lasky-Su, Jessica; Wilson, Michael R; Cedars, Marcelle; Elias, Joshua E; MacKenzie, Tippi C; DeRisi, Joseph L

Integrated single-nuclei and spatial transcriptomic profiling of human sacrococcygeal teratomas reveals heterogeneity in cellular composition and X-chromosome inactivation.

对人类骶尾部畸胎瘤进行单核和空间转录组分析,揭示了细胞组成和 X 染色体失活的异质性。

Rojas Ernesto J, Giannikou Krinio, Huang Benjamin J, Cho Soo-Jin, Cordero Marco A, Pena Deion, Vu Lan, Bagrodia Aditya, Derderian S Christopher, MacKenzie Tippi C, Laird Diana J

In utero lipid nanoparticle delivery achieves robust editing in hematopoietic stem cells.

子宫内脂质纳米颗粒递送可实现对造血干细胞的强效编辑

Worthington Atesh K, Borges Beltran, Lum Tony, Echeverri Elisa Schrader, Zada Fareha Moulana, Cordero Marco A, Kim Hyejin, Zenhausern Ryan, Celik Ozgenur, Shaw Cindy, Gutierrez-Martinez Paula, Omarova Marzhana, Blanchard Chris, Burns Sean, Cromer M Kyle, Dahlman James E, MacKenzie Tippi C

In utero hematopoietic stem cell transplantation for Fanconi anemia

子宫内造血干细胞移植治疗范可尼贫血

Swartzrock, Leah; Dib, Carla; Denis, Morgane; Willner, Hana; Ho, Katie; Haslett, Ethan; Han, Jian; Pan, Wenjing; Byrne-Steele, Miranda; Brown, Brittany; Krampf, Mark R; Girsen, Anna; Blumenfeld, Yair J; El-Sayed, Yasser Y; Roncarolo, Maria G; MacKenzie, Tippi C; Czechowicz, Agnieszka D