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Characterisation of the ocular inflammatory response to AAV reveals divergence by sex and age.
Mol Ther
January 2025
Academic Unit of Ophthalmology, Translational Health Sciences, University of Bristol, Bristol, BS8 1TD, UK; NIHR Biomedical Research Centre of Ophthalmology, Moorfields Eye Hospital, London, EC1V 2PD, UK. Electronic address:
Progress for ocular AAV gene therapy has been hindered by AAV-induced inflammation, limiting dose escalation and long-term efficacy. Broadly, the extent of inflammatory responses alters with age and sex, yet these factors are poorly represented in pre-clinical development of ocular AAV gene therapies. Here, we combined clinical imaging, flow cytometry and bulk-sequencing of sorted microglia to interrogate the longitudinal inflammatory response following intravitreal delivery of AAV2 in young (3-month), middle aged (9-month) and old (18-month) Cx3cr1-creER:R26tdTomato+/- mice of both sexes.
View Article and Find Full Text PDFBiofunctionalized patterned platform as microarray biochip to supervise delivery and expression of pDNA nanolipoplexes in stem cells via mechanotransduction.
J Nanobiotechnology
January 2025
School of Medicine, Shanghai University, Shanghai, 200444, China.
Biochips are widely applied to manipulate the geometrical morphology of stem cells in recent years. Patterned antenna-like pseudopodia are also probed to explore the influence of pseudopodia formation on gene delivery and expression on biochips. However, how the antenna-like pseudopodia affect gene transfection is unsettled and the underlying trafficking mechanism of exogenous genes in engineered single cells is not announced.
View Article and Find Full Text PDFCurrent approaches in CRISPR-Cas systems for diabetes.
Prog Mol Biol Transl Sci
January 2025
R and D, Salem Microbes Private Limited, Salem, Tamil Nadu, India. Electronic address:
In the face of advancements in health care and a shift towards healthy lifestyle, diabetes mellitus (DM) still presents as a global health challenge. This chapter explores recent advancements in the areas of genetic and molecular underpinnings of DM, addressing the revolutionary potential of CRISPR-based genome editing technologies. We delve into the multifaceted relationship between genes and molecular pathways contributing to both type1 and type 2 diabetes.
View Article and Find Full Text PDFCRISPR challenges in clinical developments.
Prog Mol Biol Transl Sci
January 2025
Department of Biochemistry, Faculty of Biological Sciences, Tarbiat Modares University, Tehran, Iran.
CRISPR-Cas (clustered regularly interspaced short palindromic repeats and associated proteins) is a novel genome editing technology with potential applications in treating diseases. Currently, its use in humans is restricted to clinical trials, although its growth rate is significant, and some have received initial FDA approval. It is crucial to examine and address the challenges for this technology to be implemented in clinical settings.
View Article and Find Full Text PDFCurrent progress in CRISPR-Cas systems for rare diseases.
Prog Mol Biol Transl Sci
January 2025
Department of Biotechnology, Faculty of Engineering and Technology, Rama University, Kanpur, Uttar Pradesh, India. Electronic address:
The groundbreaking CRISPR-Cas gene editing method permits exact genetic code alteration. The "CRISPR" DNA protects bacteria from viruses. CRISPR-Cas utilizes a guide RNA to steer the Cas enzyme to the genome's gene editing target.
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