The current landscape of nucleic acid therapeutics

JA Kulkarni, D Witzigmann, SB Thomson… - Nature …, 2021 - nature.com
The increasing number of approved nucleic acid therapeutics demonstrates the potential to
treat diseases by targeting their genetic blueprints in vivo. Conventional treatments …

Recent advances in nanoparticulate RNA delivery systems

J Witten, Y Hu, R Langer… - Proceedings of the …, 2024 - National Acad Sciences
Nanoparticle-based RNA delivery has shown great progress in recent years with the
approval of two mRNA vaccines for Severe Acute Respiratory Syndrome Coronavirus 2 …

Enzyme-catalyzed one-step synthesis of ionizable cationic lipids for lipid nanoparticle-based mRNA COVID-19 vaccines

Z Li, XQ Zhang, W Ho, F Li, M Gao, X Bai, X Xu - ACS nano, 2022 - ACS Publications
Ionizable cationic lipid-containing lipid nanoparticles (LNPs) are the most clinically
advanced non-viral gene delivery platforms, holding great potential for gene therapeutics …

Biomimetic Mineralized CRISPR/Cas RNA Nanoparticles for Efficient Tumor-Specific Multiplex Gene Editing

Y Liang, J Zhang, C Xu, J Wang, W Han, J Yang… - ACS …, 2023 - ACS Publications
CRISPR/Cas9 systems have great potential to achieve sophisticated gene therapy and cell
engineering by editing multiple genomic loci. However, to achieve efficient multiplex gene …

Nonviral In Vivo Delivery of CRISPR-Cas9 Using Protein-Agnostic, High-Loading Porous Silicon and Polymer Nanoparticles

RB Fletcher, LD Stokes, IB Kelly III, KM Henderson… - ACS …, 2023 - ACS Publications
The complexity of CRISPR machinery is a challenge to its application for nonviral in vivo
therapeutic gene editing. Here, we demonstrate that proteins, regardless of size or charge …

Quality assessment of LNP-RNA therapeutics with orthogonal analytical techniques

J Parot, D Mehn, H Jankevics, N Markova… - Journal of Controlled …, 2024 - Elsevier
The availability of analytical methods for the characterization of lipid nanoparticles (LNPs)
for in-vivo intracellular delivery of nucleic acids is critical for the fast development of …

Flotation coefficient distributions of lipid nanoparticles by sedimentation velocity analytical ultracentrifugation

H Zhao, AA Sousa, P Schuck - ACS nano, 2024 - ACS Publications
The robust characterization of lipid nanoparticles (LNPs) encapsulating therapeutics or
vaccines is an important and multifaceted translational problem. Sedimentation velocity …

Payload distribution and capacity of mRNA lipid nanoparticles

S Li, Y Hu, A Li, J Lin, K Hsieh, Z Schneiderman… - Nature …, 2022 - nature.com
Lipid nanoparticles (LNPs) are effective vehicles to deliver mRNA vaccines and
therapeutics. It has been challenging to assess mRNA packaging characteristics in LNPs …

[HTML][HTML] Particles in biopharmaceutical formulations, part 2: an update on analytical techniques and applications for therapeutic proteins, viruses, vaccines and cells

A Roesch, S Zölls, D Stadler, C Helbig… - Journal of …, 2022 - Elsevier
Particles in biopharmaceutical formulations remain a hot topic in drug product development.
With new product classes emerging it is crucial to discriminate particulate active …

Analytical Characterization of Heterogeneities in mRNA-Lipid Nanoparticles Using Sucrose Density Gradient Ultracentrifugation

A Vaidya, D Parande, N Khadse… - Analytical …, 2024 - ACS Publications
Rational design and robust formulation processes are critical for optimal delivery of mRNA
by lipid nanoparticles (LNPs). Varying degrees of heterogeneity in mRNA-LNPs can affect …