Engineered Silica Nanoparticles for Nucleic Acid Delivery DOI
Yue Zhang, Yingjie Yu, Yannan Yang

et al.

Small Methods, Journal Year: 2023, Volume and Issue: 8(3)

Published: Oct. 31, 2023

Abstract The development of nucleic acid‐based drugs holds great promise for therapeutic applications, but their effective delivery into cells is hindered by poor cellular membrane permeability and inherent instability. To overcome these challenges, vehicles are required to protect deliver acids efficiently. Silica nanoparticles (SiNPs) have emerged as promising nanovectors recently bioregulators gene due unique advantages. In this review, a summary recent advancements in the design SiNPs acid applications provided, mainly according specific type acids. First, structural characteristics working mechanisms various types introduced classified functions. Subsequently, each type, use enhancing performance biomedical summarized. tailored selected will be highlighted considering Lastly, limitations current research personal perspectives on future directions field presented. It expected opportune review provide insights burgeoning area next‐generation SiNP‐based systems.

Language: Английский

Types of RNA therapeutics DOI
Pouya Goleij, Mehregan Babamohamadi,

Aryan Rezaee

et al.

Progress in molecular biology and translational science, Journal Year: 2024, Volume and Issue: unknown, P. 41 - 63

Published: Jan. 1, 2024

Language: Английский

Citations

6
Chitosan nanocarriers for non-coding RNA therapeutics: A review DOI
Karthik Siram, S. Mohan, Induja Magesh

et al.

International Journal of Biological Macromolecules, Journal Year: 2024, Volume and Issue: 263, P. 130361 - 130361

Published: Feb. 21, 2024

Language: Английский

Citations

6
Antisense oligonucleotides as a precision therapy for developmental and epileptic encephalopathies DOI Creative Commons

Paloma García Quilón,

Greta Volpedo, Serena Cappato

et al.

CNS Neuroscience & Therapeutics, Journal Year: 2024, Volume and Issue: 30(11)

Published: Nov. 1, 2024

Developmental and epileptic encephalopathies (DEEs) comprise a complex spectrum of neurological disorders characterized by neurodevelopmental delay early-onset seizures primarily caused diverse genetic mutations. Traditional treatments have largely been symptomatic, focusing on seizure control without addressing the underlying causes. The advent gene therapy, particularly through antisense oligonucleotides (ASOs), offers promising avenue toward targeted therapeutic interventions. ASOs virtue their ability to modulate expression at mRNA level represent sophisticated approach counteract effects pathogenic This review delves into recent advancements in ASO technology, highlighting its application preclinical clinical settings for DEEs. We present evidence efficacy ameliorating disease phenotypes vitro vivo, alongside outcomes from ongoing trials. landscape DEEs is cusp significant transformation, underscored potential offer precise, personalized, that extend beyond symptomatic relief potentially rectify underpinnings these disorders.

Language: Английский

Citations

6
Hybrid nanovesicle of chimeric antigen receptor (CAR)-engineered cell-derived vesicle and drug-encapsulated liposome for effective cancer treatment DOI
Ha Eun Shin, Seung Wook Oh, Wooram Park

et al.

Journal of Industrial and Engineering Chemistry, Journal Year: 2023, Volume and Issue: 122, P. 127 - 137

Published: Feb. 27, 2023

Language: Английский

Citations

13
Splicing Modulation via Antisense Oligonucleotides in Recessive Dystrophic Epidermolysis Bullosa DOI Open Access
Stefan Hainzl, Lisa Trattner, Bernadette Liemberger

et al.

International Journal of Molecular Sciences, Journal Year: 2024, Volume and Issue: 25(2), P. 761 - 761

Published: Jan. 7, 2024

Antisense oligonucleotides (ASOs) represent an emerging therapeutic platform for targeting genetic diseases by influencing various aspects of (pre-)mRNA biology, such as splicing, stability, and translation. In this study, we investigated the potential modulating splicing pattern in recessive dystrophic epidermolysis bullosa (RDEB) patient cells carrying a frequent genomic variant (c.425A > G) that disrupts COL7A1 gene using short 2′-O-(2-Methoxyethyl) oligoribo-nucleotides (2′-MOE ASOs). COL7A1-encoded type VII collagen (C7) forms anchoring fibrils within skin are essential attachment epidermis to underlying dermis. As such, variants leading functionally impaired or absent C7 manifest form extensive blistering wounding. The severity disease warrants development novel therapies patients. c.425A G at exon 3/intron 3 junction lowers efficiency junction, resulting non-functional transcripts. However, found correct still occurs, albeit very low level, highlighting opportunity intervention reaction. We therefore screened 2′-MOE ASOs bind along target region ranging from intron 3/exon 4 their ability modulate splicing. identified capable increasing relative levels correctly spliced transcripts RT-PCR, sqRT-PCR, ddPCR. Furthermore, RDEB-derived equivalents treated with one most promising exhibited increase full-length expression its accurate deposition basement membrane zone (BMZ).

Language: Английский

Citations

4
Biomimetic nanodecoys deliver cholesterol-modified heteroduplex oligonucleotide to target dopaminergic neurons for the treatment of Parkinson's disease DOI Open Access
Si Huang, Yongjiang Li, Junyong Wu

et al.

Acta Biomaterialia, Journal Year: 2024, Volume and Issue: 177, P. 316 - 331

Published: Jan. 21, 2024

Language: Английский

Citations

4
Photochemical Stabilization of Self‐Assembled Spherical Nucleic Acids DOI Creative Commons
Sepideh Kaviani,

Haochen Bai,

T. P. Das

et al.

Small, Journal Year: 2025, Volume and Issue: unknown

Published: Jan. 10, 2025

Abstract Oligonucleotide therapeutics, including antisense oligonucleotides and small interfering RNA, offer promising avenues for modulating the expression of disease‐associated proteins. However, challenges such as nuclease degradation, poor cellular uptake, unspecific targeting hinder their application. To overcome these obstacles, spherical nucleic acids have emerged versatile tools acid delivery in biomedical applications. Our laboratory has introduced sequence‐defined DNA amphiphiles which self‐assemble aqueous solutions. Despite advantages, self‐assembled SNAs can be inherently fragile due to reliance on non‐covalent interactions fall apart biologically relevant conditions, specifically by interaction with serum Herein, this challenge is addressed introducing two methods covalent crosslinking via UV irradiation. Thymine photodimerization or disulfide at micellar interface enhance SNA stability against human albumin binding. This enhanced stability, particularly crosslinked SNAs, leads increased uptake. Furthermore, results sustained activity accessibility release therapeutic acid, along improvement unaided gene silencing. The findings demonstrate efficient stabilization through crosslinking, influencing release, ultimately, silencing activity. These studies further optimization exploration pre‐clinical, vivo studies.

Language: Английский

Citations

0
Multifunctional molecular hybrid for targeted colorectal cancer cells: Integrating doxorubicin, AS1411 aptamer, and T9/U4 ASO DOI Creative Commons
Kanpitcha Jiramitmongkon, Pichayanoot Rotkrua, Paisan Khanchaitit

et al.

PLoS ONE, Journal Year: 2025, Volume and Issue: 20(2), P. e0317559 - e0317559

Published: Feb. 13, 2025

Colorectal cancer (CRC) poses a global health challenge, with current treatments often harming both cancerous and normal cells. To improve efficacy, multifunctional drug delivery platform has been developed, integrating bioactive materials, anticancer agents, targeted recognition ligands into single molecule. This study aimed to create molecular hybrid (MH) containing doxorubicin, AS1411 aptamer, T9/U4 ASO regulate SW480 cell proliferation. The aptamer targets nucleolin, overexpressed on membranes, while inhibits human telomerase RNA activity, further hindering AS-T9/U4_MH was synthesized via oligonucleotide hybridization, followed by doxorubicin loading evaluation of its impact Binding capability this MH verified using fluorescence microscopy flow cytometry, demonstrating specific cells due nucleolin availability the surface. These findings were corroborated cytometry. exhibited anti-proliferative effects, doxorubicin-loaded system encapsulation reduced toxicity. Moreover, presence Dox within led notable reduction in hTERT vimentin expression Additionally, examination apoptotic pathways unveiled marked decrease Bcl-2 simultaneous increase Bax treated Dox-loaded AS-T9/U4_MH, indicating promoting apoptosis. shows promise as for chemotherapeutic drugs materials therapy.

Language: Английский

Citations

0
Synthetic Polymers for Drug, Gene, and Vaccine Delivery DOI Creative Commons
Yajun Xu, Jie Chen, Jianxun Ding

et al.

Polymer science & technology., Journal Year: 2025, Volume and Issue: unknown

Published: April 25, 2025

Language: Английский

Citations

0
Aptamer–ODN Chimeras: Enabling Cell-Specific ODN Targeting Therapy DOI Creative Commons

Xia Bei,

Qubo Zhu

Cells, Journal Year: 2025, Volume and Issue: 14(10), P. 697 - 697

Published: May 12, 2025

Oligonucleotides (ODNs) such as siRNA, saRNA, and miRNA regulate gene expression through a variety of molecular mechanisms show unique potential in the treatment genetic diseases rare diseases, but their clinical application is still limited by efficiency delivery system, especially problem insufficient targeting extrahepatic tissues. As homologous nucleic acid molecules, aptamers have become key tool to improve targeted ODNs. Aptamer–ODN chimeras can not only bind multiple proteins on cell surface with high specificity selectivity, they also internalize into cells. Furthermore, outperform traditional systems terms cost-effectiveness chemical modification flexibility. This review systematically summarizes origin progress aptamer–ODN chimera therapy, discusses some innovative design strategies, proposes views future direction aptamer-ODN chimeras.

Language: Английский

Citations

0