New Perspectives in Stem Cell Transplantation and Associated Therapies to Treat Retinal Diseases: From Gene Editing to 3D Bioprinting DOI
Gabrieli Bovi dos Santos,

Théo Henrique de Lima-Vasconcellos,

Marília Inês Móvio

et al.

Stem Cell Reviews and Reports, Journal Year: 2024, Volume and Issue: 20(3), P. 722 - 737

Published: Feb. 6, 2024

Language: Английский

Polymer- and lipid-based nanocarriers for ocular drug delivery: Current status and future perspectives DOI Creative Commons

Haijie Han,

Su Li, Mingyu Xu

et al.

Advanced Drug Delivery Reviews, Journal Year: 2023, Volume and Issue: 196, P. 114770 - 114770

Published: March 7, 2023

Ocular diseases seriously affect patients' vision and life quality, with a global morbidity of over 43 million blindness. However, efficient drug delivery to treat ocular diseases, particularly intraocular disorders, remains huge challenge due multiple barriers that significantly the ultimate therapeutic efficacy drugs. Recent advances in nanocarrier technology offer promising opportunity overcome these by providing enhanced penetration, increased retention, improved solubility, reduced toxicity, prolonged release, targeted loaded eyes. This review primarily provides an overview progress contemporary applications nanocarriers, mainly polymer- lipid-based treating various eye highlighting their value achieving delivery. Additionally, covers administration routes, as well prospective future developments challenges field nanocarriers for diseases.

Language: Английский

Citations

148

Inherited retinal diseases: Linking genes, disease-causing variants, and relevant therapeutic modalities DOI
Nina Schneider, Yogapriya Sundaresan, Prakadeeswari Gopalakrishnan

et al.

Progress in Retinal and Eye Research, Journal Year: 2021, Volume and Issue: 89, P. 101029 - 101029

Published: Nov. 25, 2021

Language: Английский

Citations

127

Retinitis Pigmentosa: Current Clinical Management and Emerging Therapies DOI Open Access
Xuan‐Thanh‐An Nguyen,

Lude Moekotte,

Astrid S. Plomp

et al.

International Journal of Molecular Sciences, Journal Year: 2023, Volume and Issue: 24(8), P. 7481 - 7481

Published: April 19, 2023

Retinitis pigmentosa (RP) comprises a group of inherited retinal dystrophies characterized by the degeneration rod photoreceptors, followed cone photoreceptors. As result photoreceptor degeneration, affected individuals experience gradual loss visual function, with primary symptoms progressive nyctalopia, constricted fields and, ultimately, central vision loss. The onset, severity and clinical course RP shows great variability unpredictability, most patients already experiencing some degree disability in childhood. While is currently untreatable for majority patients, significant efforts have been made development genetic therapies, which offer new hope treatment dystrophies. In this exciting era emerging gene it remains imperative to continue supporting using all available options manage their condition. Patients wide variety physical, mental social-emotional difficulties during lifetime, require timely intervention. This review aims familiarize readers management that are RP.

Language: Английский

Citations

57

Insights into eye genetics and recent advances in ocular gene therapy DOI Creative Commons
Viktória Szabó, Balázs Varsányi,

Mirella Telles Salgueiro Barboni

et al.

Molecular and Cellular Probes, Journal Year: 2025, Volume and Issue: 79, P. 102008 - 102008

Published: Jan. 18, 2025

Language: Английский

Citations

2

Structure and function of ABCA4 and its role in the visual cycle and Stargardt macular degeneration DOI
Robert S. Molday, Fabian A. Garces, Jéssica Fernandes Scortecci

et al.

Progress in Retinal and Eye Research, Journal Year: 2021, Volume and Issue: 89, P. 101036 - 101036

Published: Dec. 23, 2021

Language: Английский

Citations

60

Pericyte dysfunction and loss of interpericyte tunneling nanotubes promote neurovascular deficits in glaucoma DOI Creative Commons
Luis Alarcón-Martínez, Yukihiro Shiga,

Deborah Villafranca‐Baughman

et al.

Proceedings of the National Academy of Sciences, Journal Year: 2022, Volume and Issue: 119(7)

Published: Feb. 8, 2022

Reduced blood flow and impaired neurovascular coupling are recognized features of glaucoma, the leading cause irreversible blindness worldwide, but mechanisms underlying these defects unknown. Retinal pericytes regulate microcirculatory coordinate through interpericyte tunneling nanotubes (IP-TNTs). Using two-photon microscope live imaging mouse retina, we found reduced capillary diameter at pericyte locations in eyes with high intraocular pressure, most important risk factor to develop glaucoma. We show that IP-TNTs structurally functionally damaged by ocular hypertension, a response disrupted light-evoked coupling. Pericyte-specific inhibition excessive Ca2+ influx rescued hemodynamic responses, protected coupling, enhanced retinal neuronal function as well survival glaucomatous retinas. Our study identifies potential therapeutic targets counter pressure-related microvascular deficits, provides preclinical proof concept strategies aimed restore intrapericyte calcium homeostasis rescue autoregulatory prevent dysfunction.

Language: Английский

Citations

57

Understanding the molecular basis and pathogenesis of hereditary optic neuropathies: towards improved diagnosis and management DOI Creative Commons
Nancy J. Newman, Patrick Yu‐Wai‐Man, Valérie Biousse

et al.

The Lancet Neurology, Journal Year: 2022, Volume and Issue: 22(2), P. 172 - 188

Published: Sept. 22, 2022

Language: Английский

Citations

57

Adeno-Associated Virus (AAV) - Based Gene Therapies for Retinal Diseases: Where are We? DOI Creative Commons
Divya Ail, Hugo Malki, Emilia A. Zin

et al.

The Application of Clinical Genetics, Journal Year: 2023, Volume and Issue: Volume 16, P. 111 - 130

Published: May 1, 2023

Abstract: Owing to their small size and safety profiles, adeno-associated viruses (AAVs) have become the vector of choice for gene therapy applications in retina. In addition naturally occurring AAVs, several engineered variants with enhanced properties are being developed experimental therapeutic applications. Nonetheless, there still some challenges impeding successful application AAVs a broader range retinal therapies. The AAV particles ensures efficient tissue transduction but also limits packaging capacity few kilobases. Further, AAV's ability cross barriers is an obstacle pan-retinal outer retina tolerable doses. Lastly, despite overall safety, been recent reports immune responses eye. Hence, evaluation prediction has come be considered integral part future clinical success. This review focuses on use trials diseases, discusses developments novel strategies overcome AAVs. Keywords: therapy, virus, AAV, trials, capsid variants,

Language: Английский

Citations

28

Gene-agnostic therapeutic approaches for inherited retinal degenerations DOI Creative Commons

Molly C. John,

Joel Quinn, Monica L. Hu

et al.

Frontiers in Molecular Neuroscience, Journal Year: 2023, Volume and Issue: 15

Published: Jan. 9, 2023

Inherited retinal diseases (IRDs) are associated with mutations in over 250 genes and represent a major cause of irreversible blindness worldwide. While gene augmentation or editing therapies could address the underlying genetic small subset patients, their utility remains limited by great heterogeneity IRDs costs developing individualised therapies. Gene-agnostic therapeutic approaches target common pathogenic pathways that drive degeneration provide functional rescue vision independent cause, thus offering potential clinical benefits to all IRD patients. Here, we review key gene-agnostic approaches, including cell reprogramming replacement, neurotrophic support, immune modulation optogenetics. The relative limitations these strategies timing interventions discussed.

Language: Английский

Citations

25

The Role of Müller Cells in Diabetic Macular Edema DOI Creative Commons
Dongwei Lai, Yang Wu,

Chuhan Shao

et al.

Investigative Ophthalmology & Visual Science, Journal Year: 2023, Volume and Issue: 64(10), P. 8 - 8

Published: July 7, 2023

Diabetic macular edema (DME) is a common complication of diabetic retinopathy and the leading cause vision loss in patients. Various factors, such as metabolic disorders inflammation caused by hyperglycemia, are involved occurrence development DME, but specific mechanism still unclear. Müller cells type macroglial cell unique to fundus, distributed throughout retina, they play role retinal homeostasis. This article reviews pathological process DME research progress treatment targeting through gene therapy.

Language: Английский

Citations

24