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 …

In vivo liver targeted genome editing as therapeutic approach: progresses and challenges

C Simoni, E Barbon, AF Muro… - Frontiers in Genome …, 2024 - frontiersin.org
The liver is an essential organ of the body that performs several vital functions, including the
metabolism of biomolecules, foreign substances, and toxins, and the production of plasma …

Recent Therapeutic Gene Editing Applications to Genetic Disorders

E Deneault - Current Issues in Molecular Biology, 2024 - mdpi.com
Recent years have witnessed unprecedented progress in therapeutic gene editing,
revolutionizing the approach to treating genetic disorders. In this comprehensive review, we …

In vivo LNP-CRISPR Approaches for the Treatment of Hemophilia

JH Lee, JP Han - Molecular Diagnosis & Therapy, 2024 - Springer
Hemophilia is a genetic disorder that is caused by mutations in coagulation factor VIII
(hemophilia A) or IX (hemophilia B) genes resulting in blood clotting disorders. Despite …