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

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

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

Intrauterine enzyme replacement therapies for lysosomal storage disorders: Current developments and promising future prospects

溶酶体贮积症的宫内酶替代疗法:当前进展和充满希望的未来前景

Herzeg, Akos; Borges, Beltran; Lianoglou, Billie R; Gonzalez-Velez, Juan; Canepa, Emma; Munar, Dane; Young, Sarah P; Bali, Deeksha; Gelb, Michel H; Chakraborty, Pranesh; Kishnani, Priya S; Harmatz, Paul; Cohen, Jennifer L; MacKenzie, Tippi C