DNA tetrahedron nanoparticles service as a help carrier and adjvant of mRNA vaccine DOI Creative Commons
Huijun Liu, Xianxian Li,

Ru-Yong Yan

et al.

Journal of Translational Medicine, Journal Year: 2024, Volume and Issue: 22(1)

Published: Nov. 14, 2024

To investigate the potential of DNA nanoparticles (DNPs) as carriers and adjuvants for mRNA vaccines. Customized oligonucleotides were assembled into tetrahedra (DNA-TH), which subsequently complexed with streptavidin encoding green fluorescent protein (GFP). Various assays conducted to evaluat stability DNPs, their cellular uptake, immune activation potential, GFP transcription efficiency. P53-mutant HSC-3 cells used establish a subcutaneous xenograft tumor model explore effects DNPs in disease model. The remained stable extracellularly rapidly taken up by antigen-presenting cells. Compared naked mRNA, statistically significantly activated responses facilitated expression both vitro vivo. Immunization DNP-GFP complexes induced higher antibody titers compared mRNA. demonstrated good biocompatibility. DNP-p53 inhibited growth tumors mice p53-mutant cells, outperforming p53 blank control groups, significant difference (P < 0.05). show promise improving vaccine delivery efficacy. Further optimization these could lead highly effective broad applications.

Language: Английский

Update on Clinical Trial Endpoints in Gene Therapy Trials for Inherited Retinal Diseases DOI Open Access
J. Igoe,

Byron L. Lam,

Ninel Z. Gregori

et al.

Journal of Clinical Medicine, Journal Year: 2024, Volume and Issue: 13(18), P. 5512 - 5512

Published: Sept. 18, 2024

Inherited retinal diseases (IRDs) encompass a wide spectrum of rare conditions characterized by diverse phenotypes associated with hundreds genetic variations, often leading to progressive visual impairment and profound vision loss. Multiple natural history studies clinical trials exploring gene therapy for various IRDs are ongoing. Outcomes ophthalmic measure changes in three main categories-structural, functional, patient-focused outcomes. Since may range from congenital poor central birth affecting the peripheral retina initially progressing insidiously acuity affected late disease course, typical outcome measures such as ocular coherence tomography (OCT) imaging macula not provide adequate representation therapeutic outcomes including alterations course. Thus, alternative unique necessary assess loss vision, color night contrast sensitivity IRDs. These differences have complicated assessment IRD therapies, had design novel specialized endpoints demonstrate treatment efficacy. As engineering techniques continue advance growing investment industry accelerated approval tracks orphan conditions, must improve their assessments safety efficacy new therapies that aim come market. Here, we will an overview current approaches, review measuring function, highlight those utilized recent trials, stage 2 3 through second quarter 2024.

Language: Английский

Citations

5
Recent advances and prospects of nanoparticle-based drug delivery for diabetic ocular complications DOI Creative Commons
Siqi Wang, Hongyu Yang, Jiaying Zheng

et al.

Theranostics, Journal Year: 2025, Volume and Issue: 15(8), P. 3551 - 3570

Published: Feb. 25, 2025

Diabetes mellitus (DM) is a chronic metabolic disorder that significantly affects various organ systems. The systemic effects of DM lead to numerous complications, with ocular manifestations being particular concern due their severity and impact on quality life. Hyperglycemia-induced damage often results in range lesions, including diabetic retinopathy (DR), keratopathy, cataracts, glaucoma. These conditions impose considerable physical discomfort patients place substantial economic burden healthcare advent nanotechnology has facilitated the development innovative therapeutic strategies for managing complications. This review highlights several common complications associated DM, focusing pathogenesis treatment strategies. Emphasis placed applications potential treating

Language: Английский

Citations

0
Gene therapy shines light on congenital stationary night blindness for future cures DOI Creative Commons
Yi Zhang, Siqi Lin,

Lingqi Yu

et al.

Journal of Translational Medicine, Journal Year: 2025, Volume and Issue: 23(1)

Published: April 3, 2025

Congenital Stationary Night Blindness (CSNB) is a non-progressive hereditary eye disease that primarily affects the retinal signal processing, resulting in significantly reduced vision under low-light conditions. CSNB encompasses various subtypes, each with distinct genetic patterns and pathogenic genes. Over past few decades, gene therapy for disorders has made substantial progress; however, effective clinical therapies are yet to be discovered. With continuous advancement of gene-therapy tools, there potential these methods become treatments CSNB. Nonetheless, challenges remain treatment CSNB, including issues related delivery vectors, therapeutic efficacy, possible side effects. This article reviews diagnosis, pathogenesis, associated mutated genes discusses existing animal models, explores application technologies disorders, as well current state research on

Language: Английский

Citations

0
DNA tetrahedron nanoparticles service as a help carrier and adjvant of mRNA vaccine DOI Creative Commons
Huijun Liu, Xianxian Li,

Ru-Yong Yan

et al.

Journal of Translational Medicine, Journal Year: 2024, Volume and Issue: 22(1)

Published: Nov. 14, 2024

To investigate the potential of DNA nanoparticles (DNPs) as carriers and adjuvants for mRNA vaccines. Customized oligonucleotides were assembled into tetrahedra (DNA-TH), which subsequently complexed with streptavidin encoding green fluorescent protein (GFP). Various assays conducted to evaluat stability DNPs, their cellular uptake, immune activation potential, GFP transcription efficiency. P53-mutant HSC-3 cells used establish a subcutaneous xenograft tumor model explore effects DNPs in disease model. The remained stable extracellularly rapidly taken up by antigen-presenting cells. Compared naked mRNA, statistically significantly activated responses facilitated expression both vitro vivo. Immunization DNP-GFP complexes induced higher antibody titers compared mRNA. demonstrated good biocompatibility. DNP-p53 inhibited growth tumors mice p53-mutant cells, outperforming p53 blank control groups, significant difference (P < 0.05). show promise improving vaccine delivery efficacy. Further optimization these could lead highly effective broad applications.

Language: Английский

Citations

1