公开(公告)号：US20230357798A1

公开(公告)日：2023-11-09

申请号：US18029798

申请日：2021-10-12

申请人： THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY

发明人： Mara Pavel-Dinu ,  Matthew H. Porteus

IPC分类号： C12N15/90 ,  C12N9/22 ,  C12N15/11 ,  A61K35/28 ,  A61P37/00

CPC分类号： C12N15/907 ,  C12N9/22 ,  C12N15/11 ,  C12N15/111 ,  A61K35/28 ,  A61P37/00 ,  C12N2310/20 ,  C12N2310/321 ,  C12N2310/315 ,  C12N2320/34

摘要： The present disclosure provides methods and compositions for treating X-CGD in subjects, comprising genetically modifying cells from the subjects ex vivo by integrating a functional, codon-optimized CYBB cDNA at the endogenous CYBB locus.

公开(公告)号：US20240173355A1

公开(公告)日：2024-05-30

申请号：US18551975

申请日：2022-04-05

申请人： The Board of Trustees of the Leland Stanford Junior University

发明人： Mara Pavel-Dinu ,  Matthew H. Porteus

IPC分类号： A61K35/28 ,  A61K35/545 ,  A61P37/04 ,  C12N5/074 ,  C12N5/0789 ,  C12N9/22 ,  C12N15/11 ,  C12N15/86

CPC分类号： A61K35/28 ,  A61K35/545 ,  A61P37/04 ,  C12N5/0647 ,  C12N5/0696 ,  C12N9/22 ,  C12N15/111 ,  C12N15/86 ,  C12N2310/20 ,  C12N2750/14143

摘要： The present disclosure provides methods and compositions for treating RAG2 deficiencies in subjects, comprising genetically modifying cells from the subjects ex vivo by integrating a functional, codon-optimized RAG 2 cDNA at the endogenous RAG2 locus.

公开(公告)号：US20240093242A1

公开(公告)日：2024-03-21

申请号：US18007439

申请日：2021-08-03

申请人： The Board of Trustees of the Leland Stanford Junior University

发明人： Mara Pavel-Dinu ,  Matthew H. Porteus

IPC分类号： C12N15/90 ,  A61K35/28 ,  C12N5/0789 ,  C12N9/22 ,  C12N15/113

CPC分类号： C12N15/907 ,  A61K35/28 ,  C12N5/0647 ,  C12N9/22 ,  C12N15/1138 ,  C12N2310/20 ,  C12N2310/315 ,  C12N2310/344 ,  C12N2510/00 ,  C12N2800/80

摘要： The present disclosure provides methods and compositions for treating SCID-X1 in subjects, comprising genetically modifying cells from the subjects ex vivo by integrating a full-length, codon-optimized IL2RG cDNA at the endogenous IL2RG locus.