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

Haplotype editing with CRISPR-Cas9 as a therapeutic approach for dominant-negative missense mutations in NEFL

利用 CRISPR-Cas9 进行单倍型编辑作为治疗 NEFL 中显性负性错义突变的治疗方法

Dua, Poorvi H; Simon, Bazilco M J; Marley, Chiara B E; Feliciano, Carissa M; Watry, Hannah L; Cowan, Quinn T; Steury, Dylan; Abraham, Abin; Gilbertson, Erin N; Ramey, Grace D; Capra, John A; Conklin, Bruce R; Judge, Luke M

Leveraging human genetic variation to therapeutically target hundreds of genes with dominant & dispensable disease alleles

利用人类遗传变异,以治疗为目标,靶向数百个具有显性和非显性致病等位基因的基因。

Ramey, Grace D; Cowan, Quinn T; Saxena, Akshita G; Macklin, Bria L; Watry, Hannah L; Mei, Xiaoyue; Dierks, Philip; Judge, Luke M; Conklin, Bruce R; Capra, John A

Characterizing and controlling CRISPR repair outcomes in nondividing human cells

对非分裂人类细胞中 CRISPR 修复结果进行表征和控制

Gokul N Ramadoss,Samali J Namaganda,Manasi M Kumar,Jennifer R Hamilton,Rohit Sharma,Karena G Chow,Luke A Workley,Bria L Macklin,Mengyuan Sun,Alvin S Ha,Jia-Cheng Liu,Christof Fellmann,Hannah L Watry,Philip H Dierks,Rudra S Bose,Julianne Jin,Barbara S Perez,Cindy R Sandoval Espinoza,Madeline P Matia,Serena H Lu,Luke M Judge,Brian R Shy,Andre Nussenzweig,Britt Adamson,Niren Murthy,Jennifer A Doudna,Martin Kampmann,Bruce R Conklin

Haplotype editing with CRISPR/Cas9 as a therapeutic approach for dominant-negative missense mutations in NEFL

使用 CRISPR/Cas9 进行单倍型编辑作为 NEFL 显性负错义突变的治疗方法

Poorvi H Dua, Bazilco M J Simon, Chiara B E Marley, Carissa M Feliciano, Hannah L Watry, Dylan Steury, Abin Abraham, Erin N Gilbertson, Grace D Ramey, John A Capra, Bruce R Conklin, Luke M Judge

Neuronal DNA repair reveals strategies to influence CRISPR editing outcomes

神经元 DNA 修复揭示了影响 CRISPR 编辑结果的策略

Gokul N Ramadoss, Samali J Namaganda, Jennifer R Hamilton, Rohit Sharma, Karena G Chow, Bria L Macklin, Mengyuan Sun, Jia-Cheng Liu, Christof Fellmann, Hannah L Watry, Julianne Jin, Barbara S Perez, Cindy R Sandoval Espinoza, Madeline P Matia, Serena H Lu, Luke M Judge, Andre Nussenzweig, Britt Adam

Reversal of C9orf72 mutation-induced transcriptional dysregulation and pathology in cultured human neurons by allele-specific excision

通过等位基因特异性切除逆转培养人类神经元中 C9orf72 突变引起的转录失调和病理

Aradhana Sachdev, Kamaljot Gill, Maria Sckaff, Alisha M Birk, Olubankole Aladesuyi Arogundade, Katherine A Brown, Runvir S Chouhan, Patrick Oliver Issagholian-Lewin, Esha Patel, Hannah L Watry, Mylinh T Bernardi, Kathleen C Keough, Yu-Chih Tsai, Alec Simon Tulloch Smith, Bruce R Conklin, Claire Dudl

Functional analysis of a common BAG3 allele associated with protection from heart failure.

对与预防心力衰竭相关的常见 BAG3 等位基因进行功能分析

Perez-Bermejo Juan A, Judge Luke M, Jensen Christina L, Wu Kenneth, Watry Hannah L, Truong Annie, Ho Jaclyn J, Carter Matthew, Runyon Wendy V, Kaake Robyn M, Pulido Ernst H, Mandegar Mohammad A, Swaney Danielle L, So Po-Lin, Krogan Nevan J, Conklin Bruce R

PAX8 modulates the tumor microenvironment of high grade serous ovarian cancer through changes in the secretome

PAX8 通过分泌组的变化调节高级别浆液性卵巢癌的肿瘤微环境

Amrita Salvi, Laura R Hardy, Kimberly N Heath, Samantha Watry, Melissa R Pergande, Stephanie M Cologna, Joanna E Burdette

Allele-Specific Gene Editing Rescues Pathology in a Human Model of Charcot-Marie-Tooth Disease Type 2E

等位基因特异性基因编辑挽救了 2E 型夏科-马里-图斯病人类模型中的病理

Carissa M Feliciano, Kenneth Wu, Hannah L Watry, Chiara B E Marley, Gokul N Ramadoss, Hana Y Ghanim, Angela Z Liu, Lyandysha V Zholudeva, Todd C McDevitt, Mario A Saporta, Bruce R Conklin, Luke M Judge

Rapid, precise quantification of large DNA excisions and inversions by ddPCR

通过 ddPCR 快速、精确地定量大片段 DNA 切除和倒位

Hannah L Watry, Carissa M Feliciano, Ketrin Gjoni, Gou Takahashi, Yuichiro Miyaoka, Bruce R Conklin, Luke M Judge0