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http://dx.doi.org/10.1021/jacs.5b12653 | DOI Listing |
PLoS Biol
June 2025
Department of Metabolic Diseases, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht, The Netherlands.
Mutations in the mitochondrial genome can cause maternally inherited diseases, cancer, and aging-related conditions. Recent technological progress now enables the creation and correction of mutations in the mitochondrial genome, but it remains relatively unknown how patients with primary mitochondrial disease can benefit from this technology. Here, we demonstrate the potential of the double-stranded DNA deaminase toxin A-derived cytosine base editor (DdCBE) to develop disease models and therapeutic strategies for mitochondrial disease in primary human cells.
View Article and Find Full Text PDFChem Soc Rev
June 2025
State Key Laboratory of Flexible Electronics (LoFE), Jiangsu Key Laboratory of Smart Biomaterials and Theranostic Technology, Institute of Advanced Materials (IAM), Nanjing University of Posts & Telecommunications, Nanjing 210023, China.
Correction for 'DNA-mediated precise regulation of SERS hotspots for biosensing and bioimaging' by Jingjing Zhang , , 2025, https://doi.org/10.1039/d5cs00124b.
View Article and Find Full Text PDFSci Rep
April 2025
Division of KM Science Research, Korea Institute of Oriental Medicine, 1672 Yuseong-daero, Yuseong-gu, Daejeon, 34054, South Korea.
The LRRK2 G2019S mutation is known to have a high penetrance rate associated with Parkinson's disease (PD), prevalent across both familial and sporadic PD cases and implicated in neurodegenerative mechanisms. This mutation disrupts several key cellular processes, particularly affecting the endoplasmic reticulum and mitochondrial functions in neural stem cells (NSCs), which are crucial for protein homeostasis and energy metabolism. Although aging is a major risk factor for PD, the complex interplay between LRRK2 G2019S and aging-related cellular dysfunction in NSCs remains poorly understood.
View Article and Find Full Text PDFInfect Dis Poverty
February 2024
Background: Schistosoma japonicum is a parasitic flatworm that causes human schistosomiasis, which is a significant cause of morbidity in China, the Philippines and Indonesia. Oncomelania hupensis (Gastropoda: Pomatiopsidae) is the unique intermediate host of S. japonicum.
View Article and Find Full Text PDFCells
February 2023
Université Paris-Saclay, UVSQ, Inserm, END-ICAP, 78000 Versailles, France.
Nucleic acid-based therapeutics hold great promise for the treatment of numerous diseases, including neuromuscular disorders, such as Duchenne muscular dystrophy (DMD). Some antisense oligonucleotide (ASO) drugs have already been approved by the US FDA for DMD, but the potential of this therapy is still limited by several challenges, including the poor distribution of ASOs to target tissues, but also the entrapment of ASO in the endosomal compartment. Endosomal escape is a well recognized limitation that prevents ASO from reaching their target pre-mRNA in the nucleus.
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