Roadmap to discovery and early development of an mRNA loaded LNP formulation for liver therapeutic genome editing DOI Creative Commons
Annette Bak, Liping Zhou,

Joanna Rejman

et al.

Expert Opinion on Drug Delivery, Journal Year: 2025, Volume and Issue: unknown

Published: Jan. 11, 2025

Introduction mRNA therapeutics were a niche area in drug development before COVIDvaccines. Now they are used vaccine development, for non-viral therapeuticgenome editing, vivo chimericantigen receptor T (CAR T) celltherapies and protein replacement. mRNAis large, charged, easily degraded by nucleases. It cannot get into cells,escape the endosome, be translated to disease-modifying without adelivery system such as lipid nanoparticles (LNPs).

Language: Английский

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
Looking back, moving forward: protein corona of lipid nanoparticles DOI
Yue Gao,

Yeqi Huang,

Chuanyu Ren

et al.

Journal of Materials Chemistry B, Journal Year: 2024, Volume and Issue: 12(23), P. 5573 - 5588

Published: Jan. 1, 2024

Intelligent delivery of lipid nanoparticles can be achieved through rational design protein corona as a “troublemaker”.

Language: Английский

Citations

4
Non-viral vectors for chimeric antigen receptor immunotherapy DOI
Sandy Tretbar, Joel G. Rurik,

Even H Rustad

et al.

Nature Reviews Methods Primers, Journal Year: 2024, Volume and Issue: 4(1)

Published: Oct. 10, 2024

Language: Английский

Citations

4
The role of patient-specific variables in protein corona formation and therapeutic efficacy in nanomedicine DOI Creative Commons

Ethan Cisneros,

Brinkley Morse,

Ani Savk

et al.

Journal of Nanobiotechnology, Journal Year: 2024, Volume and Issue: 22(1)

Published: Nov. 15, 2024

Despite their potential, the adoption of nanotechnology in therapeutics remains limited, with only around eighty nanomedicines approved past 30 years. This disparity is partly due to "one-size-fits-all" approach medical design, which often overlooks patient-specific variables such as biological sex, genetic ancestry, disease state, environment, and age that influence nanoparticle behavior. Nanoparticles (NPs) must be transported through systemic, microenvironmental, cellular barriers vary across heterogeneous patient populations. Key patient-dependent properties impacting NP delivery include blood flow rates, body fat distribution, reproductive organ vascularization, hormone protein levels, immune responses, chromosomal differences. Understanding these crucial for developing effective, nanotechnologies. The formation a corona NPs upon exposure fluids significantly alters properties, affecting biodistribution, pharmacokinetics, cytotoxicity, targeting. dynamics corona, time-dependent composition soft hard coronas, depend on characteristics serum components. review highlights importance understanding different backgrounds its implications including age, state. By exploring variables, we aim advance development personalized nanomedicine, improving therapeutic efficacy outcomes.

Language: Английский

Citations

4
Roadmap to discovery and early development of an mRNA loaded LNP formulation for liver therapeutic genome editing DOI Creative Commons
Annette Bak, Liping Zhou,

Joanna Rejman

et al.

Expert Opinion on Drug Delivery, Journal Year: 2025, Volume and Issue: unknown

Published: Jan. 11, 2025

Introduction mRNA therapeutics were a niche area in drug development before COVIDvaccines. Now they are used vaccine development, for non-viral therapeuticgenome editing, vivo chimericantigen receptor T (CAR T) celltherapies and protein replacement. mRNAis large, charged, easily degraded by nucleases. It cannot get into cells,escape the endosome, be translated to disease-modifying without adelivery system such as lipid nanoparticles (LNPs).

Language: Английский

Citations

0