公开(公告)号：US20230113805A1

公开(公告)日：2023-04-13

申请号：US17905794

申请日：2021-03-11

Applicant: BRAIN BIOTECH AG

Inventor： Paul SCHOLZ ,  Uemit PUL ,  Christian ZUREK ,  Yukoh HIEI ,  Chizu YANAGIHARA ,  Hiroshi TSUKAMOTO ,  Toshihiko KOMARI

IPC: C12N9/22 ,  C12N15/11 ,  C12N15/82

Abstract: The present invention relates to a nucleic acid molecule encoding an RNA-guided DNA endonuclease, which is (a) a nucleic acid molecule encoding the RNA-guided DNA endonuclease comprising or consisting of the amino acid sequence of SEQ ID NO: 1 or 3; (b) a nucleic acid molecule comprising or consisting of the nucleotide sequence of SEQ ID NO: 2 or 4; (c) a nucleic acid molecule encoding a RNA-guided DNA endonuclease the amino acid sequence of which is at least 70% identical to the amino acid sequence of (a); preferably at least 80% identical, more preferably at least 90% identical, and most preferred at least 95% identical; (d) a nucleic acid molecule comprising or consisting of a nucleotide sequence which is at least 70% identical to the nucleotide sequence of (b), preferably at least 80% identical, more preferably at least 90% identical, and most preferred at least 95% identical; (e) a nucleic acid molecule which is degenerate with respect to the nucleic acid molecule of (d); or (f) a nucleic acid molecule corresponding to the nucleic acid molecule of any one of (a) to (d) wherein T is replaced by U.

公开(公告)号：US20250135030A1

公开(公告)日：2025-05-01

申请号：US18730505

申请日：2024-01-18

Applicant: BRAIN BIOTECH AG

Inventor： Paul SCHOLZ ,  Christian ZUREK

IPC: A61K48/00 ,  C12N9/22 ,  C12N15/11 ,  C12N15/85 ,  C12N15/90

Abstract: The present invention relates to a method for the genome editing of cells at a target locus by homology directed repair (HDR) within a cell population and concurrently enriching within said cell population the HDR-genome edited cells. The present invention also relates to a method for the selective depletion of cells comprising a target locus within a cell population.

公开(公告)号：US20230287372A1

公开(公告)日：2023-09-14

申请号：US18006324

申请日：2021-07-20

Applicant: BRAIN Biotech AG

Inventor： Paul SCHOLZ ,  Christian ZUREK ,  Michael KROHN

IPC: C12N9/22 ,  C12N15/82

CPC classification number: C12N9/22 ,  C12N15/8201 ,  C12N2310/20

Abstract: The present invention relates to a nucleic acid molecule encoding an RNA-guided DNA endonuclease, which is (a) a nucleic acid molecule encoding the RNA-guided DNA endonuclease comprising or consisting of the amino acid sequence of SEQ ID NO: 29, 1 or 3; (b) a nucleic acid molecule comprising or consisting of the nucleotide sequence of SEQ ID NO: 30, 2 or 4; (c) a nucleic acid molecule encoding a RNA-guided DNA endonuclease the amino acid sequence of which is at least 90%, preferably at least 92 %, and most preferably at least 95% identical to the amino acid sequence of (a); (d) a nucleic acid molecule comprising or consisting of a nucleotide sequence which is at least 90%, preferably at least 92%, and most preferably at least 95% identical to the nucleotide sequence of (b); (e) a nucleic acid molecule which is degenerate with respect to the nucleic acid molecule of (d); or (f) a nucleic acid molecule corresponding to the nucleic acid molecule of any one of (a) to (d) wherein T is replaced by U.