The current landscape of nucleic acid therapeutics
The increasing number of approved nucleic acid therapeutics demonstrates the potential to
treat diseases by targeting their genetic blueprints in vivo. Conventional treatments …
treat diseases by targeting their genetic blueprints in vivo. Conventional treatments …
Recent advances in nanoparticulate RNA delivery systems
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 …
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
Ionizable cationic lipid-containing lipid nanoparticles (LNPs) are the most clinically
advanced non-viral gene delivery platforms, holding great potential for gene therapeutics …
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 …
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 …
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 …
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
The robust characterization of lipid nanoparticles (LNPs) encapsulating therapeutics or
vaccines is an important and multifaceted translational problem. Sedimentation velocity …
vaccines is an important and multifaceted translational problem. Sedimentation velocity …
Payload distribution and capacity of mRNA lipid nanoparticles
Lipid nanoparticles (LNPs) are effective vehicles to deliver mRNA vaccines and
therapeutics. It has been challenging to assess mRNA packaging characteristics in LNPs …
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 …
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 …
by lipid nanoparticles (LNPs). Varying degrees of heterogeneity in mRNA-LNPs can affect …