Search Results - gene+editing

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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

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

The First Curative Treatment of Phenylketonuria Using Genetic Engineering Approaches

The base editing of phenylalanine hydroxylase enzyme in the liver to restore phenylalanine metabolism and prevent neurotoxic effects.

Published: 10/28/2024
Inventor(s):
Kiran Musunuru, Xiao Wang, Dominique Brooks
Keywords(s):
Gene Editing, Gene Therapy, Genetic Disorders, Neurodegenerative Diseases, Neurology
Category(s):
Technology Classifications > Therapeutics

LNP Delivery of Gene-Editing Machinery to Cure Hereditary Tyrosinemia Type 1g

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Published: 7/10/2024
Inventor(s):
Kiran Musunuru, Madelynn Whittaker, Xiao Wang, William Peranteau, Cara Berkowitz, Ana Dumitru
Keywords(s):
Gene Editing, Genetic Disorders, Obesity, Metabolic Disease and Diabetes
Category(s):
Technology Classifications > Therapeutics

Strategies for Mitigating Acute Side Effects of Lung-Targeted Nanocarriers

Approaches and Design Techniques to Alleviate Coagulation-Related Acute Side Effects of Lung-Targeted Nanocarriers

Published: 6/26/2024
Inventor(s):
Jacob Brenner, Vladimir Muzykantov, Jacob Myerson, Oscar Marcos Contreras, Marco Zamora, Serena Omo-Lamai
Keywords(s):
Bioengineering, Drug Delivery, Gene Editing, Gene Therapy, Nanotechnology, Platform Technology, Pulmonary
Category(s):
Technology Classifications > Therapeutics

Cholesterol Analogues Incorporated Into LNPs For Enhanced mRNA Delivery

Hydroxycholesterols are used to formulate lipid nanoparticles (LNPs) for enhanced mRNA delivery into T cells.

Published: 2/6/2024
Inventor(s):
Michael Mitchell, Margaret Billingsley, Savan Patel, Xuexiang Han, Hanwen Zhang
Keywords(s):
Bioengineering, Cell Therapy, Drug Delivery, Gene Editing, Gene Therapy, Genetic Disorders, Immunology, Immunoncology, Immunotherapy, Inflammation, Nanotechnology, Platform Technology
Category(s):
Technology Classifications > Therapeutics

Amniotic Fluid Stabilized Lipid Nanoparticles (LNPs) for In Utero Intra-Amniotic mRNA Delivery

Nanoparticles for potent encapsulation and delivery of therapeutic molecules for prenatal fetal therapy in the amniotic sac

Published: 2/7/2024
Inventor(s):
Michael Mitchell, Kelsey Swingle, Margaret Billingsley, William Peranteau
Keywords(s):
Bioengineering, Cell Therapy, Gene Editing, Gene Therapy, Genetic Disorders, Immunology, Nanotechnology, Orphan Disease, Platform Technology
Category(s):
Technology Classifications > Therapeutics

Ligand-Tethered Lipid Nanoparticles for Targeted RNA Delivery to Treat Liver Fibrosis

A new class of anisimide-functionalized lipids that enables targeted delivery of lipid nanoparticles (LNPs) to activated fibroblasts in the liver.

Published: 2/7/2024
Inventor(s):
Michael Mitchell, Xuexiang Han
Keywords(s):
Bioengineering, Drug Delivery, Gene Editing, Gene Therapy, Genetic Disorders, Immunology, Immunoncology, Platform Technology, Regenerative Medicine
Category(s):
Technology Classifications > Therapeutics, Technology Classifications > Vaccines