Bioengineering strategies for restoring vision

J Cehajic-Kapetanovic, MS Singh, E Zrenner… - Nature biomedical …, 2023 - nature.com
Late-stage retinal degenerative disease involving photoreceptor loss can be treated by
optogenetic therapy, cell transplantation and retinal prostheses. These approaches aim to …

Peptide-guided lipid nanoparticles deliver mRNA to the neural retina of rodents and nonhuman primates

M Herrera-Barrera, RC Ryals, M Gautam, A Jozic… - Science …, 2023 - science.org
Lipid nanoparticle (LNP)–based mRNA delivery holds promise for the treatment of inherited
retinal degenerations. Currently, LNP-mediated mRNA delivery is restricted to the retinal …

[HTML][HTML] Therapy approaches for Stargardt disease

E Piotter, ME McClements, RE MacLaren - Biomolecules, 2021 - mdpi.com
Despite being the most prevalent cause of inherited blindness in children, Stargardt disease
is yet to achieve the same clinical trial success as has been achieved for other inherited …

[HTML][HTML] Non-viral delivery of CRISPR/Cas cargo to the retina using nanoparticles: Current possibilities, challenges, and limitations

A Salman, A Kantor, ME McClements, G Marfany… - Pharmaceutics, 2022 - mdpi.com
The discovery of the CRISPR/Cas system and its development into a powerful genome
engineering tool have revolutionized the field of molecular biology and generated …

[HTML][HTML] Tropism of AAV vectors in photoreceptor-like cells of human iPSC-derived retinal organoids

ME McClements, H Steward, W Atkin… - … Vision Science & …, 2022 - iovs.arvojournals.org
Purpose: To expand the use of human retinal organoids from induced pluripotent stem cells
(iPSCs) as an in vitro model of the retina for assessing gene therapy treatments, it is …

New perspectives in stem cell transplantation and associated therapies to treat retinal diseases: from gene editing to 3D bioprinting

G Bovi dos Santos, TH de Lima-Vasconcellos… - Stem Cell Reviews and …, 2024 - Springer
Inherited and non-inherited retinopathies can affect distinct cell types, leading to progressive
cell death and visual loss. In the last years, new approaches have indicated exciting …

In Silico CRISPR-Cas-Mediated Base Editing Strategies for Early-Onset, Severe Cone–Rod Retinal Degeneration in Three Crumbs homolog 1 Patients, including the …

H Shamsnajafabadi, M Kaukonen, JS Bellingrath… - Genes, 2024 - mdpi.com
Pathogenic variants in the Crumbs homolog 1 (CRB1) gene lead to severe, childhood-onset
retinal degeneration leading to blindness in early adulthood. There are no approved …

Robotising vitreoretinal surgeries

H Mi, RE MacLaren, J Cehajic-Kapetanovic - Eye, 2024 - nature.com
The use of robotic surgery in ophthalmology has been shown to offer many potential
advantages to current surgical techniques. Vitreoretinal surgery requires complex …

Nanotechnology in retinal disease: Current concepts and future directions

NJ Mehta, SN Mehta - Journal of Ocular Pharmacology and …, 2024 - liebertpub.com
The retina is one of the most complex and extraordinary human organs affected by genetic,
metabolic, and degenerative diseases, resulting in blindness for∼ 1.3 million people in the …

[HTML][HTML] Minicircle delivery to the neural retina as a gene therapy approach

F Staurenghi, ME McClements, A Salman… - International Journal of …, 2022 - mdpi.com
Non-viral gene therapy has the potential to overcome several shortcomings in viral vector-
based therapeutics. Methods of in vivo plasmid delivery have developed over recent years …