Results 181 to 190 of about 34,085 (286)

An Open‐Label Phase 1b Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Clinical Activity of ANX005 in Patients with Huntington's Disease

Movement Disorders, EarlyView.
Complement activation is implicated in Huntington's disease; ANX005 is a potent inhibitor of component C1q. ANX005 exhibited a generally manageable safety profile with rapid reduction in C1q in the cerebrospinal fluid. Functional ability on composite Unified Huntington's Disease Rating Scale and total functional capacity was maintained, with potential ...
Rajeev Kumar, Pinky Agarwal, Karen Anderson, Daniel Claassen, Marissa Dean, Andrew Duker, Burton Scott, Benjamin Hoehn, Ann Mongan, Ping Lin, Ellen Cahir‐McFarland, Lori Taylor, Glenn Morrison, Ted Yednock, Sanjay Keswani, Henk‐Andre Kroon   +15 more
wiley   +1 more source

In vivo adenine base editing of mutant Galc gene ameliorates Krabbe disease progression

Genome Medicine
Background Krabbe disease (KD) is caused by mutation of the galactosylceramidase (GALC) gene, leading to deficient sphingolipid metabolism, which is essential for functional myelination.
Bae-Geun Nam, Jung Hwa Seo, Sung-Ah Hong, Ju-Hee Kim, Minju Kang, Geneva Rose Notario, Yoontaik Hong, Seunghee Cho, Sangsu Bae, Sung-Rae Cho   +9 more
doaj   +1 more source

Improving the specificity of adenine base editor using high-fidelity Cas9 [PDF]

, 2019
Ruisha Hong, Sidi Ma, Feng Wang
openalex   +1 more source

Deciphering the soybean root rot pathogen Phytophthora sojae: Signaling pathways, transcriptional regulation, and effector biology

New Plant Protection, EarlyView.
This review highlights how Phytophthora sojae utilizes unconventional lineage‐specific signaling networks, including unique G protein‐coupled receptor fusions, expanded kinases, and rapidly evolving effectors, to infect soybeans. Understanding these divergent molecular paradigms reveals critical vulnerabilities in this destructive pathogen, offering ...
Min Qiu, Xiaoyi Zhang, Bo Yang, Wenwu Ye, Yuanchao Wang   +4 more
wiley   +1 more source

The Mycobacterium tuberculosis Rv0132c Gene Product Mtb‐FGD2 Can Act as an F420‐Dependent Glucose Dehydrogenase

Proteins: Structure, Function, and Bioinformatics, EarlyView.
ABSTRACT The role of the cell envelope‐associated Rv0132c/FGD2 from Mycobacterium tuberculosis has long been a subject of debate. Importantly, FGD2 is found only in pathogenic mycobacteria, making it a potential drug target. While some suggest it functions as a glucose‐6‐phosphate dehydrogenase, others propose it acts instead as an F420‐dependent ...
Adewale V. Aderemi, Matthew Snee, Richard B. Tunnicliffe, Linus O. Johanissen, Matthew J. Cliff, Colin W. Levy, Derren J. Heyes, Marina Golovanova, Thomas A. Jowitt, Sam Hay, Andrew W. Munro, Jonathan P. Waltho, David Leys   +12 more
wiley   +1 more source

An all-in-one AAV vector for cardiac-specific gene silencing by an adenine base editor [PDF]

Zhanzhao Liu, Luzi Yang, Yuhan Yang, Jiting Li, Zhan Chen, Congting Guo, Qianhao Guo, Qiuxuan Li, Yueshen Sun, Dongyu Zhao, Xiaomin Hu, Fei Gao, Yuxuan Guo   +12 more
openalex   +1 more source

Author Correction: Targeted exon skipping with AAV-mediated split adenine base editors [PDF]

, 2019
Jackson Winter, Alan Luu, Michael Gapinske, Sony Manandhar, Shraddha Shirguppe, Wendy S. Woods, Jun S. Song, Pablo Pérez‐Piñera   +7 more
openalex   +1 more source

Engineered domain-inlaid Nme2Cas9 adenine base editors with increased on-target DNA editing and targeting scope [PDF]

, 2023
Ding Zhao, Xun Gao, Jiale Zhou, Jinze Li, Yuqiang Qian, Di Wang, Wenchao Niu, Tao Zhang, Mingyang Hu, Haoyang Xiong, Liangxue Lai, Zhanjun Li   +11 more
openalex   +1 more source

RNA degradomics and proteomics reveal the mechanism of dsProsβ1‐mediated proteasome targeting in Psylliodes chrysocephala

Pest Management Science, EarlyView.
RNA degradomics revealed dsProsβ1‐derived siRNA‐mediated mRNA cleavage events, mainly at uracil‐guanine and adenine‐adenine pairs. Proteasome inhibition via dsProsβ1 increased mitochondrial and cytoskeletal proteins while reducing translation‐related and mRNA‐binding proteins.
Doga Cedden, Gözde Güney, Michael Rostás, Stefan Scholten   +3 more
wiley   +1 more source

617 Peptide-mediated delivery of adenine base editors to rhesus monkey airway epithelia

, 2022
K. Kulhankova, S. Traore, S. Hallée, X. Cheng, V. Caron, C. Lauvaux, X. Barbeau, M. Harvey, J. Roberge, A. Tarantal, G. Newby, D. Liu, D. Guay, P. McCray   +13 more
openalex   +1 more source