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Guanidyl-rich highly branched poly(β-amino ester)s for the delivery of dual CRISPR ribonucleoprotein for efficient large DNA fragment deletion.
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Charles Institute of Dermatology, School of Medicine, University College Dublin, Dublin, Ireland. Electronic address:
Gene editing technologies, particularly clustered regularly interspersed short palindromic repeats (CRISPR) and CRISPR-associated (Cas) proteins, have revolutionized the ability to modify gene sequences in living cells for therapeutic purposes. Delivery of CRISPR/Cas ribonucleoprotein (RNP) is preferred over its DNA and RNA formats in terms of gene editing effectiveness and low risk of off-target events. However, the intracellular delivery of RNP poses significant challenges and necessitates the development of non-viral vectors.
View Article and Find Full Text PDFAdeno-Associated Virus 8 and 9 Myofibre Type/Size Tropism Profiling Reveals Therapeutic Effect of Microdystrophin in Canines.
J Cachexia Sarcopenia Muscle
February 2025
Department of Molecular Microbiology and Immunology, School of Medicine, University of Missouri, Columbia, Missouri, USA.
Background: Adeno-associated virus (AAV) 8 and 9 are in clinical trials for treating neuromuscular diseases such as Duchenne muscular dystrophy (DMD). Muscle consists of myofibres of different types and sizes. However, little is known about the fibre type and fibre size tropism of AAV in large mammals.
View Article and Find Full Text PDFAdvanced phasing techniques in congenital skin diseases.
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December 2024
Department of Dermatology, Faculty of Medicine and Graduate School of Medicine, Hokkaido University, Sapporo, Japan.
Phasing, the process of determining which alleles at different loci on homologous chromosomes belong together on the same chromosome, is crucial in the diagnosis and management of autosomal recessive diseases. Advances in long-read sequencing technologies have significantly enhanced our ability to accurately determine haplotypes. This review discusses the application of low-coverage long-read sequencing, nanopore Cas9-guided long-read sequencing, and adaptive sampling in phasing, highlighting their utility in complex clinical scenarios.
View Article and Find Full Text PDF[Impact of manual-physical correction method on aerobic metabolism processes in patients with essential arterial hypertension].
Vopr Kurortol Fizioter Lech Fiz Kult
December 2024
S.I. Spasokukotsky Moscow Centre for Research and Practice in Medical Rehabilitation, Restorative and Sports Medicine of the Moscow Healthcare Department, Moscow, Russia.
Unlabelled: Essential arterial hypertension (EAH) is a chronic non-communicable disease (CNCD), that develops in parallel with other pathologies of the CNCD group, the presence of which is promoted by hypodynamia with consequent disturbance of aerobic energy supply processes. These disorders include, in particular, degenerative-dystrophic processes of the locomotor system, comprising of the cervical spine. In turn, development of these processes can additionally worsen hemodynamics with disturbance of the oxygen transfer processes.
View Article and Find Full Text PDFChoroid plexus aging: structural and vascular insights from the HCP-aging dataset.
Fluids Barriers CNS
December 2024
Department of Radiology, NYU Grossman School of Medicine, 660 First Ave, Room 405, New York, NY, 10016, USA.
Article Synopsis
- The choroid plexus (ChP) is vital for producing cerebrospinal fluid and clearing metabolic waste, with its enlargement linked to aging and neurodegenerative diseases like Alzheimer's.
- Researchers analyzed data from 641 individuals aged 36-90, using advanced imaging techniques to examine age-related changes in ChP's volume, blood flow, and diffusion metrics.
- Findings revealed significant age-related changes in the ChP, including increased volume and decreased blood flow, indicating a potential compensatory mechanism in response to reduced perfusion.
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