[HTML][HTML] Exploring the promising potential of induced pluripotent stem cells in cancer research and therapy

M Chehelgerdi, F Behdarvand Dehkordi… - Molecular Cancer, 2023 - Springer
The advent of iPSCs has brought about a significant transformation in stem cell research,
opening up promising avenues for advancing cancer treatment. The formation of cancer is a …

[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 …

[PDF][PDF] CRB1 is required for recycling by RAB11A+ vesicles in human retinal organoids

TM Buck, PMJ Quinn, LP Pellissier, AA Mulder… - Stem cell reports, 2023 - cell.com
CRB1 gene mutations can cause early-or late-onset retinitis pigmentosa, Leber congenital
amaurosis, or maculopathy. Recapitulating human CRB1 phenotypes in animal models has …

[HTML][HTML] Characterization and AAV-mediated CRB gene augmentation in human-derived CRB1KO and CRB1KOCRB2+/− retinal organoids

N Boon, X Lu, CA Andriessen, M Orlovà… - … Therapy-Methods & …, 2023 - cell.com
The majority of patients with mutations in CRB1 develop either early-onset retinitis
pigmentosa as young children or Leber congenital amaurosis as newborns. The cause for …

Human CRB1 and CRB2 form homo-and heteromeric protein complexes in the retina

IF Stehle, JA Imventarza, F Woerz… - Life Science …, 2024 - life-science-alliance.org
Crumbs homolog 1 (CRB1) is one of the key genes linked to retinitis pigmentosa and Leber
congenital amaurosis, which are characterized by a high clinical heterogeneity. The Crumbs …

[HTML][HTML] AAV capsid bioengineering in primary human retina models

A Westhaus, SS Eamegdool, M Fernando… - Scientific Reports, 2023 - nature.com
Adeno-associated viral (AAV) vector-mediated retinal gene therapy is an active field of both
pre-clinical as well as clinical research. As with other gene therapy clinical targets, novel …

[HTML][HTML] Retinal organoids with X-linked retinoschisis RS1 (E72K) mutation exhibit a photoreceptor developmental delay and are rescued by gene augmentation …

C Duan, C Ding, X Sun, S Mao, Y Liang, X Liu… - Stem Cell Research & …, 2024 - Springer
Background X-linked juvenile retinoschisis (XLRS) is an inherited disease caused by RS1
gene mutation, which leads to retinal splitting and visual impairment. The mechanism of RS1 …

[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 …

Classical and Innovative Evidence for Therapeutic Strategies in Retinal Dysfunctions

L Caruso, M Fields, E Rimondi, G Zauli… - International Journal of …, 2024 - mdpi.com
The human retina is a complex anatomical structure that has no regenerative capacity. The
pathogenesis of most retinopathies can be attributed to inflammation, with the activation of …

[HTML][HTML] Diverse functions and pathogenetic role of Crumbs in retinopathy

X Zhou, L Zhao, C Wang, W Sun, B Jia, D Li… - Cell Communication and …, 2024 - Springer
The Crumbs protein (CRB) family plays a crucial role in maintaining the apical–basal
polarity and integrity of embryonic epithelia. The family comprises different isoforms in …