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Benjamin P. Kleinstiver

massachusetts general hospital
H指数:41
被引数:1.4万

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Toward the clinical application of long-read sequencing in repeat-expansion disorders

长读测序在重复序列扩增疾病中的临床应用展望

2026-08-200
PRE
Mechanistic machine learning for prediction of prime editing outcomes

机器学习方法预测碱基编辑结果机制

2026-08-120
OAAI
Extracellular vesicles-mediated delivery of SpCas9 RNPs for therapeutic gene editing in Spinocerebellar Ataxia Type 3

外泌体介导的SpCas9核糖核蛋白递送,用于脊髓小脑共济失调3型的治疗性基因编辑

2026-03-090
OAAI
Nucleotide-resolution mapping of regulatory elements via allelic readout of tiled base editing

通过平铺碱基编辑的等位基因读出调控元件的核苷酸分辨率定位

2026-03-030
OAAI
Customizing CRISPR–Cas PAM specificity with protein language models

使用蛋白质语言模型定制crispr-cas PAM特异性

2026-02-020
PRE
An opportune time for targeted brain arteriovenous malformation therapy

靶向脑动静脉畸形治疗的有利时机

2025-12-010
PRE
Treatment of a severe vascular disease using a bespoke CRISPR–Cas9 base editor in mice

使用定制的CRISPR–Cas9碱基编辑器治疗小鼠中的严重血管疾病

2025-09-110
PRE
Non-invasive detection of allele-specific CRISPR-SaCas9-KKH disruption of TOR1A DYT1 allele in a xenograft mouse model

非侵入性检测异种移植小鼠模型中TOR1A DYT1等位基因特异的CRISPR-SaCas9-KKH破坏

2025-01-010
OAAI
CRISPR targeting of mmu-miR-21a through a single adeno-associated virus vector prolongs survival of glioblastoma-bearing mice

通过单一腺相关病毒载体对mmu-miR-21a进行CRISPR靶向治疗可延长胶质母细胞瘤小鼠的生存期

2025-01-011
OAAI
Myocardial infarction augments sleep to limit cardiac inflammation and damage
Nature
IF48.5
2024-10-300
PRE
High-fidelity PAMless base editing of hematopoietic stem cells to treat chronic granulomatous disease

造血干细胞的高保真无PAMless碱基编辑治疗慢性肉芽肿病

2024-10-160
PRE
Splice modulators target PMS1 to reduce somatic expansion of the Huntington's disease-associated CAG repeat
2024-04-122
OAAI
In situ architecture of Opa1-dependent mitochondrial cristae remodeling
2024-01-159
OAAI
Optimization of base editors for the functional correction of SMN2 as a treatment for spinal muscular atrophy

用于SMN2功能矫正的碱基编辑器的优化,以治疗脊髓性肌萎缩症

2023-12-065
OA
PAM-flexible genome editing with an engineered chimeric Cas9
2023-10-0411
OAAI