FMR1 Disorders: Basics of Biology and Therapeutics in Development DOI Creative Commons

Drew A. Gillett,

Helene Tigro,

Yuan Wang

et al.

Cells, Journal Year: 2024, Volume and Issue: 13(24), P. 2100 - 2100

Published: Dec. 18, 2024

Fragile X Syndrome (FXS) presents with a constellation of phenotypes, including trouble regulating emotion and aggressive behaviors, disordered sleep, intellectual impairments, atypical physical development. Genetic study the chromosome revealed that substantial repeat expansion 5′ end gene fragile messenger ribonucleoprotein 1 (FMR1) promoted DNA methylation and, consequently, silenced expression FMR1. Further analysis proved shorter expansions in FMR1 also manifested disease at later stages life. Treatment therapy options do exist, but they only manage symptoms. Up to now, no cure for disorders exists. In this review, we aim provide an overview biology latest research focused on developing therapeutic interventions can potentially prevent and/or reverse FXS.

Language: Английский

Expanding the role of exosomes in drug, biomolecule, and nanoparticle delivery DOI
Ongun Mehmet Saka, Devrim Demir Dora, Güneş Kibar

et al.

Life Sciences, Journal Year: 2025, Volume and Issue: 368, P. 123499 - 123499

Published: Feb. 22, 2025

Language: Английский

Citations

1
Engineered hsa‐miR‐455‐3p‐Abundant Extracellular Vesicles Derived from 3D‐Cultured Adipose Mesenchymal Stem Cells for Tissue‐Engineering Hyaline Cartilage Regeneration DOI
Jianguo Chen, Enchong Zhang, Yingying Wan

et al.

Advanced Healthcare Materials, Journal Year: 2024, Volume and Issue: 13(18)

Published: March 20, 2024

Efforts are made to enhance the inherent potential of extracellular vesicles (EVs) by utilizing 3D culture platforms and engineered strategies for functional cargo-loading. Three distinct types adipose mesenchymal stem cells-derived EVs (ADSCs-EVs) successfully isolated consisting porous gelatin methacryloyl (PG), PG combined with sericin (PG/SerMA), or chondroitin sulfate (PG/ChSMA). These correspond PG-EVs, PG/SerMA-EVs, PG/ChSMA-EVs, respectively. Unique microRNA (miRNA) profiles observed in each type ADSCs-EVs. Notably, PG-EVs encapsulate higher levels hsa-miR-455-3p deliver more chondrocytes, which results activation hsa-miR-455-3p/PAK2/Smad2/3 axis subsequent hyaline cartilage regeneration. Furthermore, functionality is optimized through strategies, including agomir/lentivirus transfection, electroporation, Exo-Fect transfection. referred as Agomir-EVs, Lentivirus-EVs, Electroporation-EVs, Exo-Fect-EVs, respectively, ranked based on their efficacy encapsulating hsa-miR-455-3p, delivering promoting formation via axis. Exo-Fect-EVs exhibit highest efficiency. Collectively, conditions have an impact miRNA regeneration capabilities The findings provide valuable insights into mechanisms underlying promotion

Language: Английский

Citations

5
Extracellular Vesicle Spherical Nucleic Acids DOI Creative Commons
Hao Chen,

Qiaojiao Ding,

Lin Li

et al.

JACS Au, Journal Year: 2024, Volume and Issue: 4(6), P. 2381 - 2392

Published: May 30, 2024

Extracellular vesicles (EVs) are naturally occurring secreted by cells that can transport cargo between cells, making them promising bioactive nanomaterials. However, due to the complex and heterogeneous biological characteristics, a method for robust EV manipulation efficient delivery is still lacking. Here, we developed novel class of extracellular vesicle spherical nucleic acid (EV-SNA) nanostructures with scalability, programmability, cellular delivery. EV-SNA was constructed through simple hydrophobic coassembly natural EVs cholesterol-modified oligonucleotides be stable 1 month at room temperature. Based on programmable shells, respond AND logic gates achieve assembly manipulation. Importantly, from wide range sources EV, enhancing capability nearly 10–20 times. Compared artificial liposomal SNA, endogenous exhibited better biocompatibility more effective antisense in hard-to-transfect primary stem cells. Additionally, deliver functional immune regulation. As material form, may provide modular framework paradigm EV-based applications drug delivery, disease treatment, nanovaccines, other fields.

Language: Английский

Citations

4
Exosomes in the Chemoresistance of Glioma: Key Point in Chemoresistance DOI Creative Commons

Xu Guo,

Haozhe Piao,

Rui Sui

et al.

Journal of Cellular and Molecular Medicine, Journal Year: 2025, Volume and Issue: 29(4)

Published: Feb. 1, 2025

ABSTRACT Gliomas are the most ordinary primary virulent brain tumours and commonly used clinical treatments include tumour resection, radiation therapy chemotherapy. Although significant progress has been made in recent years progression‐free survival (PFS) overall (OS) for patients with high‐grade gliomas, prognosis remains poor. Chemoresistance refers to phenomenon of decreased sensitivity cells drugs, resulting reduced or ineffective drug efficacy, is an important cause failure Exosomes, a type extracellular vesicle, secreted by cancer various stromal microenvironment (TME) transfer their inclusions cells, increasing chemoresistance. Furthermore, depletion exosomes reverses certain detrimental effects on metabolism restores chemotherapeutic agents. Here, we summarised correlation between resistance agents glioma patients, mechanisms action involved value. We aimed afford new thoughts research, diagnosis intervention chemoresistance patients.

Language: Английский

Citations

0
iLight2: A near‐infrared optogenetic tool for gene transcription with low background activation DOI
Mikhail Baloban, L.A. Kasatkina, Vladislav V. Verkhusha

et al.

Protein Science, Journal Year: 2024, Volume and Issue: 33(5)

Published: April 22, 2024

Abstract Optogenetic tools (OTs) operating in the far‐red and near‐infrared (NIR) region offer advantages for light‐controlling biological processes deep tissues spectral multiplexing with fluorescent probes OTs acting visible range. However, many NIR suffer from background activation darkness. Through shortening linkers, we engineered a novel OT, iLight2, which exhibits significantly reduced activity darkness, thereby increasing light‐to‐dark contrast. The resultant optimal configuration of iLight2 components suggests molecular mechanism action. Using biliverdin reductase knock‐out mouse model, show that advanced performance primary cells vivo . Efficient light‐controlled cell migration wound healing cellular model demonstrates possibility using therapy and, overall, positions it as valuable addition to OT toolkit gene transcription applications.

Language: Английский

Citations

3
From conventional to cutting-edge: Exosomes revolutionizing nano-drug delivery systems DOI

Huiyang Fu,

Yinfeng Chen,

Qingyao Fu

et al.

Chemical Engineering Journal, Journal Year: 2024, Volume and Issue: unknown, P. 156685 - 156685

Published: Oct. 1, 2024

Language: Английский

Citations

2
Exosomes for protein and peptide drug delivery DOI

Dhwani Rana,

Nimeet Desai, Sagar Salave

et al.

Elsevier eBooks, Journal Year: 2024, Volume and Issue: unknown, P. 305 - 327

Published: Nov. 29, 2024

Citations

0
FMR1 Disorders: Basics of Biology and Therapeutics in Development DOI Creative Commons

Drew A. Gillett,

Helene Tigro,

Yuan Wang

et al.

Cells, Journal Year: 2024, Volume and Issue: 13(24), P. 2100 - 2100

Published: Dec. 18, 2024

Fragile X Syndrome (FXS) presents with a constellation of phenotypes, including trouble regulating emotion and aggressive behaviors, disordered sleep, intellectual impairments, atypical physical development. Genetic study the chromosome revealed that substantial repeat expansion 5′ end gene fragile messenger ribonucleoprotein 1 (FMR1) promoted DNA methylation and, consequently, silenced expression FMR1. Further analysis proved shorter expansions in FMR1 also manifested disease at later stages life. Treatment therapy options do exist, but they only manage symptoms. Up to now, no cure for disorders exists. In this review, we aim provide an overview biology latest research focused on developing therapeutic interventions can potentially prevent and/or reverse FXS.

Language: Английский

Citations

0