Deciphering the role of nanocarrier-based nucleic acid delivery to the brain for the management of neurodegenerative disorders DOI

Subham Panigrahy,

Anupama Sikder,

Etikala Amulya

et al.

Journal of Drug Delivery Science and Technology, Journal Year: 2023, Volume and Issue: 92, P. 105325 - 105325

Published: Dec. 30, 2023

Language: Английский

Recent Progress in the Endosomal Escape Mechanism and Chemical Structures of Polycations for Nucleic Acid Delivery DOI
Mohit J. Mehta, Hyun Jin Kim,

Sung Been Lim

et al.

Macromolecular Bioscience, Journal Year: 2024, Volume and Issue: 24(4)

Published: Jan. 16, 2024

Abstract Nucleic acid‐based therapies are seeing a spiralling surge. Stimuli‐responsive polymers, especially pH‐responsive ones, gaining widespread attention because of their ability to efficiently deliver nucleic acids. These polymers can be synthesized and modified according target requirements, such as delivery sites the nature In this regard, endosomal escape mechanism polymer–nucleic acid complexes (polyplexes) remains topic considerable interest owing various plausible mechanisms. This review describes current progress in polyplexes state‐of‐the‐art chemical designs for polymers. The importance is also discussed dissociation constant (i.e., p K ) designing new generation along with assays monitor quantify behavior. Further, use machine learning addressed prediction polymer design find novel structures pH responsiveness. will facilitate advanced efficient delivery.

Language: Английский

Citations

23

Advancements and challenges in developing in vivo CAR T cell therapies for cancer treatment DOI Creative Commons
Thuỳ Anh Bùi, Haoqi Mei, Rui Sang

et al.

EBioMedicine, Journal Year: 2024, Volume and Issue: 106, P. 105266 - 105266

Published: Aug. 1, 2024

The Chimeric Antigen Receptor (CAR) T cell therapy has emerged as a ground-breaking immunotherapeutic approach in cancer treatment. To overcome the complexity and high manufacturing cost associated with current ex vivo CAR products, alternative strategies to produce cells directly body have been developed recent years. These involve direct infusion of genes via engineered nanocarriers or viral vectors generate situ. This review offers comprehensive overview advancements development cell-targeted generation Additionally, it identifies challenges method potential these issues.

Language: Английский

Citations

18

Strategies and mechanisms for endosomal escape of therapeutic nucleic acids DOI Creative Commons

Melina Grau,

Ernst Wagner

Current Opinion in Chemical Biology, Journal Year: 2024, Volume and Issue: 81, P. 102506 - 102506

Published: Aug. 1, 2024

Despite impressive recent establishment of therapeutic nucleic acids as drugs and vaccines, their broader medical use is impaired by modest performance in intracellular delivery. Inefficient endosomal escape presents a major limitation responsible for inadequate cytosolic cargo release. Depending on the carrier, this barrier can strongly limit or even abolish acid Different strategies hypothesized mechanisms are reviewed.

Language: Английский

Citations

13

Nucleic acid drugs: recent progress and future perspectives DOI Creative Commons

Xiaoyi Sun,

Sarra Setrerrahmane,

Chencheng Li

et al.

Signal Transduction and Targeted Therapy, Journal Year: 2024, Volume and Issue: 9(1)

Published: Nov. 29, 2024

Language: Английский

Citations

13

Current RNA strategies in treating cardiovascular diseases DOI Creative Commons
Shirley Pei Shan Chia, Jeremy Kah Sheng Pang, Boon-Seng Soh

et al.

Molecular Therapy, Journal Year: 2024, Volume and Issue: 32(3), P. 580 - 608

Published: Jan. 29, 2024

Cardiovascular disease (CVD) continues to impose a significant global health burden, necessitating the exploration of innovative treatment strategies. Ribonucleic acid (RNA)-based therapeutics have emerged as promising avenue address complex molecular mechanisms underlying CVD pathogenesis. We present comprehensive review current state RNA in context CVD, focusing on diverse modalities that bring about transient or permanent modifications by targeting different stages biology central dogma. Considering immense potential therapeutics, we identified common gene targets could serve interventions for prevalent Mendelian caused single mutations, well acquired CVDs developed over time due various factors. These offer opportunities develop RNA-based treatments tailored specific genetic and pathways, presenting novel precise approach pathogenesis both types cardiovascular conditions. Additionally, discuss challenges associated with delivery strategies achieve targeted system. This highlights combat paving way future advancements therapeutics.

Language: Английский

Citations

9

Protein-based delivery systems for RNA delivery DOI

Haichao Zhu,

Hong Luo,

Ruilong Chang

et al.

Journal of Controlled Release, Journal Year: 2023, Volume and Issue: 363, P. 253 - 274

Published: Sept. 28, 2023

Language: Английский

Citations

17

Protein-based nanoparticles for therapeutic nucleic acid delivery DOI Creative Commons
Feyisayo Eweje, Michelle L. Walsh,

Kiran Ahmad

et al.

Biomaterials, Journal Year: 2024, Volume and Issue: 305, P. 122464 - 122464

Published: Jan. 2, 2024

Language: Английский

Citations

8

Recent advances in liposomes and peptide-based therapeutics for glioblastoma treatment DOI Creative Commons

M.A. Jourdain,

Joël Eyer

Journal of Controlled Release, Journal Year: 2024, Volume and Issue: 376, P. 732 - 752

Published: Oct. 31, 2024

In the context of glioblastoma treatment, penetration drugs is drastically limited by blood-brain-barrier (BBB). Emerging therapies have focused on field therapeutic peptides for their excellent BBB targeting properties that promote a deep tumor penetration. Peptide-based strategies are also renowned abilities driving cargo such as liposomal system allowing an active receptors overexpressed GBM cells. This review provides detailed description internalization mechanisms specific homing and penetrating well latest in vitro/in vivo studies liposomes functionalized with them. The purpose this to summarize selection promising pre-clinical results demonstrate advantages nanosystem, including increase cell targeting, triggering drug accumulation thus strong antitumor effect. Aware early stage these studies, many challenges need be overcome peptide-directed liposome at clinical level. particular, lack suitable production, difficulty characterize nanosystem competition leaded antibodies.

Language: Английский

Citations

5

Revival of Bioengineered Proteins as Carriers for Nucleic Acids DOI Creative Commons

David C. Scherer,

Michael Burger, Jean‐Christophe Leroux

et al.

Bioconjugate Chemistry, Journal Year: 2024, Volume and Issue: 35(5), P. 561 - 566

Published: April 15, 2024

ADVERTISEMENT RETURN TO ARTICLES ASAPViewpointNEXTRevival of Bioengineered Proteins as Carriers for Nucleic AcidsDavid SchererDavid SchererInstitute Pharmaceutical Sciences, Department Chemistry and Applied Biosciences, ETH Zurich, Zurich 8093, SwitzerlandMore by David Scherer, Michael Burger*Michael BurgerInstitute Switzerland*[email protected]More Burger, Jean-Christophe Leroux*Jean-Christophe LerouxInstitute Lerouxhttps://orcid.org/0000-0001-5601-1292Cite this: Bioconjugate Chem. 2024, XXXX, XXX, XXX-XXXPublication Date (Web):April 15, 2024Publication History Received22 February 2024Accepted1 April 2024Published online15 2024https://doi.org/10.1021/acs.bioconjchem.4c00079© 2024 The Authors. Published American Chemical Society. This publication is licensed under CC-BY 4.0. License Summary*You are free to share (copy redistribute) this article in any medium or format adapt (remix, transform, build upon) the material purpose, even commercially within parameters below:Creative Commons (CC): a Creative license.Attribution (BY): Credit must be given creator.View full license*DisclaimerThis summary highlights only some key features terms actual license. It not license has no legal value. Carefully review before using these materials. Open Access indicated. Learn MoreArticle Views-Altmetric-Citations-LEARN ABOUT THESE METRICSArticle Views COUNTER-compliant sum text downloads since November 2008 (both PDF HTML) across all institutions individuals. These metrics regularly updated reflect usage leading up last few days.Citations number other articles citing article, calculated Crossref daily. Find more information about citation counts.The Altmetric Attention Score quantitative measure attention that research received online. Clicking on donut icon will load page at altmetric.com with additional details score social media presence article. how calculated. Share Add toView InAdd Full Text ReferenceAdd Description ExportRISCitationCitation abstractCitation referencesMore Options onFacebookTwitterWechatLinked InRedditEmail (2 MB) Get e-AlertscloseSUBJECTS:Endosomal escape,Gene delivery,Genetics,Nucleic acids,Peptides proteins e-Alerts

Language: Английский

Citations

4

Charge-guided masking of a membrane-destabilizing peptide enables efficient endosomal escape for targeted intracellular delivery of proteins DOI Creative Commons
Yan Zhao,

Haolin Jiang,

Hang Chen

et al.

Acta Pharmaceutica Sinica B, Journal Year: 2024, Volume and Issue: 14(10), P. 4478 - 4492

Published: June 26, 2024

Intracellular delivery of biologicals such as peptides, proteins, and nucleic acids presents a great opportunity for innovative therapeutics. However, the endosome entrapment remains major bottleneck in intracellular biomacromolecules, largely limiting their therapeutic potential. Here, we converted cell-penetrating peptide (CPP), low molecular weight protamine (LMWP), to endosomal escape peptides (EEPs) by masking LMWP with pH-responsive counter-ionic peptide. The resulting masked CPPs (mLMWP mLMWP2) effectively promoted peptide/protein cargoes from endosomes into cytoplasm. Consequential lysosome repair lysophagy were initiated upon endolysosomal leakage. Minimal reactive oxygen species (ROS) elevation or cell death was observed. Based on mLMWP2, constructed an protein system containing antibody targeting module, mLMWP2 desired cargo. With HER2-targeting system, efficiently translocated cyclization recombination enzyme (Cre) BH3-interacting domain agonist (BID) cytosol HER2

Language: Английский

Citations

4