公开(公告)号：US20240252682A1

公开(公告)日：2024-08-01

申请号：US18590275

申请日：2024-02-28

Applicant: Flagship Pioneering Innovations VI, LLC

Inventor： Robert Charles Altshuler ,  Anne Helen Bothmer ,  Daniel Raymond Chee ,  Cecilia Giovanna Silvia Cotta-Ramusino ,  Kyusik Kim ,  Randi Michelle Kotlar ,  Gregory David McAllister ,  Ananya Ray ,  Nathaniel Roquet ,  Carlos Sanchez ,  Barrett Ethan Steinberg ,  William Edward Salomon ,  Robert James Citorik ,  William Querbes ,  Luciano Henrique Apponi ,  Zhan Wang ,  Yanfang Fu ,  Daniel Gene Abernathy ,  Michael Christopher Holmes

IPC: A61K48/00 ,  A61P7/06 ,  C12N9/22 ,  C12N15/11 ,  C12N15/85

CPC classification number: A61K48/005 ,  A61P7/06 ,  C12N9/22 ,  C12N15/111 ,  C12N15/85 ,  C12N2310/20

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 sickle cell disease (SCD) are described.