Manufacturing Uniform Cerebral Organoids for Neurological Disease Modeling and Drug Evaluation DOI Creative Commons
Hyowon Hong, Yesl Jun, Sae‐Bom Yoon

и другие.

Biomaterials Research, Год журнала: 2024, Номер 28

Опубликована: Янв. 1, 2024

Human cerebral organoids are promising tools for investigating brain development and the pathogenesis underlying neurological disorders. To use drug effectiveness safety screening, dispensed into each well must be prepared under precisely same conditions as cells. Despite decades of extensive research on approaches to improve organoid generation, various challenges remain, such low yields heterogeneity in size differentiation both within between batches. Here, we newly established uniform (UCOs) derived from induced pluripotent stem cells by optimizing performing real-time monitoring telencephalic marker expression. These exhibited morphological uniformity consistent expression FOXG1 during differentiation, with high productivity. Moreover, UCOs faithfully recapitulated early corticogenesis, concomitant establishment neuroepithelial populations, cortical plate neurons, glial Furthermore, systematically developed neural networks excitatory inhibitory electrophysiological signals when exposed neurotransmission blockers. Neurodevelopmental disease models manifested neurite outgrowth defects, which could ameliorated targeted treatment. We propose an advanced platform variations reproducibility modeling diseases.

Язык: Английский

Exploring the promising potential of induced pluripotent stem cells in cancer research and therapy DOI Creative Commons
Matin Chehelgerdi,

Fereshteh Behdarvand Dehkordi,

Mohammad Chehelgerdi

и другие.

Molecular Cancer, Год журнала: 2023, Номер 22(1)

Опубликована: Ноя. 28, 2023

The advent of iPSCs has brought about a significant transformation in stem cell research, opening up promising avenues for advancing cancer treatment. formation is multifaceted process influenced by genetic, epigenetic, and environmental factors. offer distinctive platform investigating the origin cancer, paving way novel approaches to treatment, drug testing, tailored medical interventions. This review article will provide an overview science behind iPSCs, current limitations challenges iPSC-based therapy, ethical social implications, comparative analysis with other types also discuss applications tumorigenesis, future tumorigenesis highlight successful case studies utilizing research. conclusion summarize advancements made research importance continued investment iPSC unlock full potential these cells.

Язык: Английский

Процитировано

67
Drug evaluation platform based on non-destructive and real-time in situ organoid fate state monitoring by graphene field-effect transistor DOI
Meng Tian,

Jinsong Wei,

Enguang Lv

и другие.

Chemical Engineering Journal, Год журнала: 2024, Номер 498, С. 155355 - 155355

Опубликована: Авг. 30, 2024

Язык: Английский

Процитировано

13
AAV-RPGR Gene Therapy Rescues Opsin Mislocalisation in a Human Retinal Organoid Model of RPGR-Associated X-Linked Retinitis Pigmentosa DOI Open Access

Paul E. Sladen,

Arifa Naeem,

Toyin Adefila-Ideozu

и другие.

International Journal of Molecular Sciences, Год журнала: 2024, Номер 25(3), С. 1839 - 1839

Опубликована: Фев. 2, 2024

Variants within the Retinitis Pigmentosa GTPase regulator (RPGR) gene are predominant cause of X-Linked (XLRP), a common and severe form inherited retinal disease. XLRP is characterised by progressive degeneration loss photoreceptors, leading to visual and, ultimately, bilateral blindness. Unfortunately, there no effective approved treatments for RPGR-associated XLRP. We sought investigate efficacy RPGRORF15 supplementation using clinically relevant construct in human RPGR-deficient organoids (ROs). Isogenic RPGR knockout (KO)-induced pluripotent stem cells (IPSCs) were generated established CRISPR/Cas9 editing methods targeting RPGR. RPGR-KO isogenic wild-type IPSCs differentiated into ROs utilised test adeno associated virus (AAV) (AAV-RPGR) clinical vector construct. The transduction AAV-RPGR successfully restored mRNA protein expression localisation photoreceptor connecting cilium rod cone photoreceptors. Vector-derived demonstrated equivalent levels glutamylation WT ROs. In addition, treatment with rhodopsin ROs, reducing mislocalisation outer nuclear layer. These data provide mechanistic insights functional potency support previously reported Phase I/II trial positive results this patients XLRP, which currently being tested III trial.

Язык: Английский

Процитировано

11
Patient-derived organoids in precision cancer medicine DOI Creative Commons
Le Tong, Weiyingqi Cui, Boya Zhang

и другие.

Med, Год журнала: 2024, Номер unknown

Опубликована: Сен. 1, 2024

Язык: Английский

Процитировано

11
Advances in the Generation of Constructed Cardiac Tissue Derived from Induced Pluripotent Stem Cells for Disease Modeling and Therapeutic Discovery DOI Creative Commons
Truman J. Roland, Kunhua Song

Cells, Год журнала: 2024, Номер 13(3), С. 250 - 250

Опубликована: Янв. 29, 2024

The human heart lacks significant regenerative capacity; thus, the solution to failure (HF) remains organ donation, requiring surgery and immunosuppression. demand for constructed cardiac tissues (CCTs) model treat disease continues grow. Recent advances in induced pluripotent stem cell (iPSC) manipulation, CRISPR gene editing, 3D tissue culture have enabled a boom iPSC-derived CCTs (iPSC-CCTs) with diverse types architecture. Compared 2D-cultured cells, iPSC-CCTs better recapitulate biology, demonstrating potential advance modeling, drug discovery, medicine, though could benefit from methods faithfully mimic physiology electrophysiology. Here, we summarize future developments vascularization, immunization, maturation of study therapy.

Язык: Английский

Процитировано

6
Revealing the clinical potential of high-resolution organoids DOI
Jihoon Ko, Sujin Hyung,

Sunghun Cheong

и другие.

Advanced Drug Delivery Reviews, Год журнала: 2024, Номер 207, С. 115202 - 115202

Опубликована: Фев. 8, 2024

Язык: Английский

Процитировано

6
Progress of organoid platform in cardiovascular research DOI Creative Commons

Xingchao Du,

Hao Jia,

Yuan Chang

и другие.

Bioactive Materials, Год журнала: 2024, Номер 40, С. 88 - 103

Опубликована: Июнь 9, 2024

Язык: Английский

Процитировано

6
Advances in Microfluidic Technologies in Organoid Research DOI
Haitao Liu, Zhongqiao Gan,

Xinyuan Qin

и другие.

Advanced Healthcare Materials, Год журнала: 2023, Номер 13(21)

Опубликована: Дек. 22, 2023

Organoids have emerged as major technological breakthroughs and novel organ models that revolutionized biomedical research by recapitulating the key structural functional complexities of their in vivo counterparts. The combination organoid systems microfluidic technologies has opened new frontiers engineering offers great opportunities to address current challenges existing broaden applications. In this review, features organoids, including origins, development, design principles, limitations, are described. Then recent progress integrating organoids into is highlighted, involving microarrays for high-throughput manipulation, microreactors hydrogel scaffold fabrication, chips culture. nascent microfluidics lie ahead accelerate disease studies, drug screening, regenerative medicine also discussed. Finally, future perspectives development advanced platforms modified building with higher fidelity standardization envisioned.

Язык: Английский

Процитировано

13
Case report: Whole exome sequencing identifies a novel variant in the HPRT1 gene in a male with developmental delay DOI Creative Commons
Haoyang Zheng, Gui Chen, Tingting Wang

и другие.

Frontiers in Genetics, Год журнала: 2025, Номер 16

Опубликована: Фев. 28, 2025

Lesch-Nyhan syndrome (LNS, OMIM #300322) is a rare X-linked genetic disorder caused by variants in the HPRT1 gene, which codes for Hypoxanthine-guanine phosphoribosyltransferase (HGPRT). gene disrupt normal purine metabolism, leading to involvement of multiple organ systems, primarily characterized hyperuricemia, dystonia, and neurological abnormalities, makes LNS clinically heterogeneous diagnostically challenging. Here, we report case 27-year-old Chinese male exhibiting severe lower limb motor disorders, intellectual development delay. Blood tests showed hyperuricemia whole exome sequencing (WES) identified novel hemizygous variant (NM-000194.3) gene: c.104T > C exon 2, respectively. Bioinformatics techniques indicated that may activity HGPRT. According clinical presentation, diagnostic examination, WES results, patient was finally diagnosed with LNS. This study previously unreported pathogenic gene. Although no curative therapy currently available at present, definite diagnosis its etiology great significance counseling family planning.

Язык: Английский

Процитировано

0
Inner Ear Organoids: Strengths and Limitations DOI Creative Commons
Giulia Pianigiani, Marta Roccio

Journal of the Association for Research in Otolaryngology, Год журнала: 2024, Номер 25(1), С. 5 - 11

Опубликована: Фев. 9, 2024

Abstract Inner ear organoids derived from differentiation of human pluripotent stem cells have recently gained momentum as tools to study inner development and developmental defects. An additional exciting aspect about this technology is represented by its translational potential, specifically, the use validate therapeutics for hearing balance restoration on human/patient-specific cells. This latter will be briefly discussed here including opportunities current limitations.

Язык: Английский

Процитировано

4