Magnetic‐Guided Delivery of Antisense Oligonucleotides for Targeted Transduction in Multiple Retinal Explant and Organoid Models DOI Creative Commons

Xiuhong Ye,

Sihui Chen, Wei Xiong

и другие.

Advanced Science, Год журнала: 2025, Номер unknown

Опубликована: Апрель 25, 2025

Abstract Antisense oligonucleotide (ASO) therapy holds promise in gene but faces challenges due to poor delivery efficiency and limited evaluation models. This investigation employs magnetic nanoparticles (MNPs) augment the of ASOs. It assesses their distribution therapeutic efficacy across various models, including retinal explants from mice macaques or human inner ear organoids. Retinal both monkeys are methodically arranged expose ganglion cell layer (GCL) photoreceptor (PL). MNPs markedly enhanced penetration targeting ASOs, resulting a 60% accumulation GCL 72% photoreceptors. Furthermore, an vitro biomimetic model neuroretina‐RPE/choroid‐sclera complex is developed examine ASO under dynamic flow conditions. Moreover, utilization MNP‐assisted ASO‐Cy3 transfection within organoids, increase positively transfected cells 70%, respectively. Here, for first time, MNP‐explant‐organoid platform carried out promotion efficiency, screening targeted delivery. development paves way investigating novel strategies diseases.

Язык: Английский

Retinal Organoids: Advances in Generation, Development, and Applications – From Stem Cells to Disease Modeling and Regenerative Medicine DOI Creative Commons

R. Mousavi,

Fatemeh Naghshnejad,

Fereshteh Karamali

и другие.

IntechOpen eBooks, Год журнала: 2025, Номер unknown

Опубликована: Янв. 31, 2025

Retinal diseases, often caused by the degeneration of light-sensitive cells, pose a significant global health challenge. While current treatments are limited, cell therapy, involving transplantation healthy offers hope. Recent advancements in stem technology have enabled creation 3D retinal organoids, which closely mimic structure and function human retina. These organoids provide valuable platform for studying development, modeling testing potential therapies. By understanding molecular mechanisms underlying eye formation disease progression, researchers can identify develop targeted treatments. hold promise revolutionizing field regenerative medicine vision research, offering hope restoring improving lives individuals affected diseases.

Язык: Английский

Процитировано

0

Retinal Organoids: Innovative Tools for Understanding Retinal Degeneration DOI Open Access
Nadia Galindo‐Cabello, Estefanía Caballano‐Infantes, Gregorio Benites

и другие.

International Journal of Molecular Sciences, Год журнала: 2025, Номер 26(7), С. 3263 - 3263

Опубликована: Апрель 1, 2025

Retinal degenerative diseases (RDDs) comprise diverse genetic and phenotypic conditions that cause progressive retinal dysfunction cell loss, leading to vision impairment or blindness. Most RDDs lack appropriate animal models for their study, which affects understanding disease mechanisms delays the progress of new treatment development. Recent advances in stem engineering, omics, organoid technology are facilitating research into there no previously existing models. The development organoids produced from human cells has impacted study as well vitro diseases, opening possibilities applications regenerative medicine, drug discovery, precision medicine. In this review, we recapitulate RDD, mentioning some main pathways underlying neurodegeneration can be studied these models, limitations future challenges rapidly advancing field.

Язык: Английский

Процитировано

0

Magnetic‐Guided Delivery of Antisense Oligonucleotides for Targeted Transduction in Multiple Retinal Explant and Organoid Models DOI Creative Commons

Xiuhong Ye,

Sihui Chen, Wei Xiong

и другие.

Advanced Science, Год журнала: 2025, Номер unknown

Опубликована: Апрель 25, 2025

Abstract Antisense oligonucleotide (ASO) therapy holds promise in gene but faces challenges due to poor delivery efficiency and limited evaluation models. This investigation employs magnetic nanoparticles (MNPs) augment the of ASOs. It assesses their distribution therapeutic efficacy across various models, including retinal explants from mice macaques or human inner ear organoids. Retinal both monkeys are methodically arranged expose ganglion cell layer (GCL) photoreceptor (PL). MNPs markedly enhanced penetration targeting ASOs, resulting a 60% accumulation GCL 72% photoreceptors. Furthermore, an vitro biomimetic model neuroretina‐RPE/choroid‐sclera complex is developed examine ASO under dynamic flow conditions. Moreover, utilization MNP‐assisted ASO‐Cy3 transfection within organoids, increase positively transfected cells 70%, respectively. Here, for first time, MNP‐explant‐organoid platform carried out promotion efficiency, screening targeted delivery. development paves way investigating novel strategies diseases.

Язык: Английский

Процитировано

0