Advancements in small molecule drug design: A structural perspective

K Wu, E Karapetyan, J Schloss, J Vadgama, Y Wu - Drug Discovery Today, 2023 - Elsevier
In this review, we outline recent advancements in small molecule drug design from a
structural perspective. We compare protein structure prediction methods and explore the …

[HTML][HTML] Clinical and molecular aspects of C2orf71/PCARE in retinal diseases

M Zufiaurre-Seijo, J García-Arumí, A Duarri - International Journal of …, 2023 - mdpi.com
Mutations in the photoreceptor-specific C2orf71 gene (also known as photoreceptor cilium
actin regulator protein PCARE) cause autosomal recessive retinitis pigmentosa type 54 and …

[HTML][HTML] Pluripotent stem cell-derived models of retinal disease: Elucidating pathogenesis, evaluating novel treatments, and estimating toxicity

M Kurzawa-Akanbi, N Tzoumas… - Progress in Retinal and …, 2024 - Elsevier
Blindness poses a growing global challenge, with approximately 26% of cases attributed to
degenerative retinal diseases. While gene therapy, optogenetic tools, photosensitive …

[HTML][HTML] Addressing Key Questions in Organoid Models: Who, Where, How, and Why?

M Gómez-Álvarez, M Agustina-Hernández… - International Journal of …, 2023 - mdpi.com
Organoids are three-dimensional cellular structures designed to recreate the biological
characteristics of the body's native tissues and organs in vitro. There has been a recent …

Pathways controlling neurotoxicity and proteostasis in mitochondrial complex I deficiency

V Nithianandam, S Sarkar… - Human Molecular …, 2024 - academic.oup.com
Neuromuscular disorders caused by dysfunction of the mitochondrial respiratory chain are
common, severe and untreatable. We recovered a number of mitochondrial genes, including …

A historical perspective of macroautophagy regulation by biochemical and biomechanical stimuli

N Dupont, A Claude‐Taupin, P Codogno - FEBS letters, 2024 - Wiley Online Library
Macroautophagy is a lysosomal degradative pathway for intracellular macromolecules,
protein aggregates, and organelles. The formation of the autophagosome, a double …

[HTML][HTML] Application of patient-derived induced pluripotent stem cells and organoids in inherited retinal diseases

Y Liang, X Sun, C Duan, S Tang, J Chen - Stem Cell Research & Therapy, 2023 - Springer
Inherited retinal diseases (IRDs) can induce severe sight-threatening retinal degeneration
and impose a considerable economic burden on patients and society, making efforts to cure …

Resilience to diabetic retinopathy

A Serikbaeva, Y Li, S Ma, D Yi, A Kazlauskas - Progress in Retinal and Eye …, 2024 - Elsevier
Chronic elevation of blood glucose at first causes relatively minor changes to the neural and
vascular components of the retina. As the duration of hyperglycemia persists, the nature and …

[HTML][HTML] Retinal Ciliopathies and Potential Gene Therapies: A Focus on Human iPSC-Derived Organoid Models

A McDonald, J Wijnholds - International Journal of Molecular Sciences, 2024 - mdpi.com
The human photoreceptor function is dependent on a highly specialised cilium. Perturbation
of cilial function can often lead to death of the photoreceptor and loss of vision. Retinal …

[HTML][HTML] Cell-cell interaction in the pathogenesis of inherited retinal diseases

X Du, AG Butler, HY Chen - Frontiers in Cell and Developmental …, 2024 - frontiersin.org
The retina is part of the central nervous system specialized for vision. Inherited retinal
diseases (IRD) are a group of clinically and genetically heterogenous disorders that lead to …