Navigating the intricate in-vivo journey of lipid nanoparticles tailored for the targeted delivery of RNA therapeutics: a quality-by-design approach

E Haghighi, SS Abolmaali, A Dehshahri… - Journal of …, 2024 - Springer
RNA therapeutics, such as mRNA, siRNA, and CRISPR–Cas9, present exciting avenues for
treating diverse diseases. However, their potential is commonly hindered by vulnerability to …

Lipid nanoparticles with PEG-variant surface modifications mediate genome editing in the mouse retina

M Gautam, A Jozic, GLN Su, M Herrera-Barrera… - Nature …, 2023 - nature.com
Ocular delivery of lipid nanoparticle (LNPs) packaged mRNA can enable efficient gene
delivery and editing. We generated LNP variants through the inclusion of positively charged …

[HTML][HTML] mRNA vaccine in gastrointestinal tumors: Immunomodulatory effects and immunotherapy

A Zhang, Q Ji, X Sheng, H Wu - Biomedicine & Pharmacotherapy, 2023 - Elsevier
Gastrointestinal tumors remain a significant healthcare burden worldwide, necessitating the
development of innovative therapeutic strategies. mRNA vaccines have emerged as a …

Lipid Nanoparticle (LNP) Delivery Carrier-Assisted Targeted Controlled Release mRNA Vaccines in Tumor Immunity

L Wu, X Li, X Qian, S Wang, J Liu, J Yan - Vaccines, 2024 - mdpi.com
In recent years, lipid nanoparticles (LNPs) have attracted extensive attention in tumor
immunotherapy. Targeting immune cells in cancer therapy has become a strategy of great …

Delivery of nucleic acid based genome editing platforms via lipid nanoparticles: Clinical applications

R Masarwy, L Stotsky-Oterin, A Elisha… - Advanced Drug Delivery …, 2024 - Elsevier
CRISPR/Cas technology presents a promising approach for treating a wide range of
diseases, including cancer and genetic disorders. Despite its potential, the translation of …

Topical gene editing therapeutics using lipid nanoparticles:'gene creams' for genetic skin diseases?

I Guri-Lamce, Y AlRokh, Y Kim… - British Journal of …, 2024 - academic.oup.com
Patients living with inherited skin diseases have benefited from recent advances in DNA
sequencing technologies that provide new or improved diagnostics. However, developing …

[HTML][HTML] Lipid Nanoparticles Enable Efficient In Vivo DNA Knock-In via HITI-Mediated Genome Editing

J Hirose, E Aizawa, S Yamamoto, M Xu, S Iwai… - Biomolecules, 2024 - mdpi.com
In vivo genome editing holds great therapeutic potential for treating monogenic diseases by
enabling precise gene correction or addition. However, improving the efficiency of delivery …

Integrating Prime Editing and Cellular Reprogramming as Novel Strategies for Genetic Cardiac Disease Modeling and Treatment

B Yao, Z Lei, MAFV Gonçalves, JPG Sluijter - Current Cardiology Reports, 2024 - Springer
Purpose of review This review aims to evaluate the potential of CRISPR-based gene editing
tools, particularly prime editors (PE), in treating genetic cardiac diseases. It seeks to answer …

When mRNA meets gene editing

W Li, C Wang, Y Lu - Nano Research, 2024 - Springer
The critical challenge of gene therapy lies in delivering gene editing agents. Compared with
DNA, while RNA is less stable and more accessible to degrade, it comes with the benefit of …

Recent Advances in Drug Delivery

AK Salem - The AAPS Journal, 2024 - Springer
In this special collection, a review on delivery strategies for RNA therapies is presented by
Dr. Oh. The review discusses different strategies employed in the design and development …