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Targeted In Utero Therapeutics Delivery System for Precise Gene Editing
Targeted lipid nanoparticles (LNPs) that can deliver mRNA-based gene editing therapeutics to hematopoietic stem cells (HSCs) in utero Problem: Monogenic blood diseases, including sickle cell disease and / thalassemia, are among the most prevalent genetic conditions worldwide, causing significant pediatric morbidity with complications like painful...
Published: 1/2/2025   |   Inventor(s): Rohan Palanki, Michael Mitchell, William Peranteau
Keywords(s): Bioengineering, Gene Editing, Gene Therapy, Platform Technology
Category(s): Technology Classifications > Therapeutics