Publication number: 20240093186
Abstract: The disclosure provides, e.g., compositions, systems, and methods for targeting, editing, modifying, or manipulating a host cell's genome at one or more locations in a DNA sequence in a cell, tissue, or subject. Gene modifying systems for treating cystic fibrosis, e.g., in subjects having a mutation resulting in F508del, are described.
Type:
Application
Filed:
September 20, 2023
Publication date:
March 21, 2024
Inventors:
Robert Charles Altshuler, Anne Helen Bothmer, Daniel Raymond Chee, Cecilia Giovanna Silvia Cotta-Ramusino, Kyusik Kim, Randi Michelle Kotlar, Gregory David McAllister, Aamir Mir, Ananya Ray, Nathaniel Roquet, Carlos Sanchez, Barrett Ethan Steinberg, Robert James Citorik, William Edward Salomon, William Querbes, Luciano Henrique Apponi, Zhan Wang