公开(公告)号：US11193164B2

公开(公告)日：2021-12-07

申请号：US15989100

申请日：2018-05-24

Applicant: The Regents of the University of California ,  Ludwig Institute for Cancer Research Ltd

Inventor： Paul Mischel ,  Prashant Mali ,  Vineet Bafna ,  Kristen Turner ,  Viraj Deshpande ,  Doruk Beyter

IPC: C12Q1/6841 ,  C12Q1/6886

Abstract: Provided herein are, inter alia, methods and compositions to detect, monitor and treat cancer, wherein the cancer includes amplified extrachromosomal oncogenes. The methods are useful for personalized treatment and exploit differential expression of amplified extrachromosomal oncogenes in cancer cells versus healthy cells.

公开(公告)号：US20240392303A1

公开(公告)日：2024-11-28

申请号：US18786394

申请日：2024-07-26

Applicant: The Regents of the University of California

Inventor： Prashant Mali ,  Dhruva Katrekar

IPC: C12N15/115 ,  A61K38/00 ,  A61P21/00 ,  C12N9/78 ,  C12N15/113

Abstract: Aspects of the disclosure relate to a gene therapy approach for diseases, disorders, or conditions caused by mutation in the stop codon utilizing modified tRNA. At least 10-15% of all genetic diseases, including muscular dystrophy (e.g. Duchene muscular dystrophy), some cancers, beta thalassemia, Hurler syndrome, and cystic fibrosis, fall into this category. Not to be bound by theory, it is believed that this approach is safer than CRISPR approaches due to minimal off-target effects and the lack of genome level changes.

公开(公告)号：US11932856B2

公开(公告)日：2024-03-19

申请号：US16490494

申请日：2018-03-02

Applicant: The Regents of the University of California

Inventor： Prashant Mali ,  Dhruva Katrekar

IPC: C12N15/113 ,  A61P21/00 ,  C12N9/78 ,  C12N15/115 ,  A61K38/00

CPC classification number: C12N15/115 ,  A61P21/00 ,  C12N9/78 ,  C12Y305/04 ,  A61K38/00 ,  C12N15/113 ,  C12N15/1137 ,  C12N2310/20 ,  C12N2310/531 ,  C12N2320/34

Abstract: Aspects of the disclosure relate to a gene therapy approach for diseases, disorders, or conditions caused by mutation in the stop codon utilizing modified tRNA. At least 10-15% of all genetic diseases, including muscular dystrophy (e.g. Duchene muscular dystrophy), some cancers, beta thalassemia, Hurler syndrome, and cystic fibrosis, fall into this category. Not to be bound by theory, it is believed that this approach is safer than CRISPR approaches due to minimal off-target effects and the lack of genome level changes.

公开(公告)号：US20230313231A1

公开(公告)日：2023-10-05

申请号：US18025169

申请日：2021-09-08

Applicant: The Regents of the University of California

Inventor： Prashant Mali ,  Dhruva Katrekar ,  Nathan Palmer

IPC: C12N15/87 ,  C12N9/78 ,  C12N9/22 ,  C12N15/11 ,  C12N15/86

CPC classification number: C12N15/87 ,  C12N9/78 ,  C12Y305/04004 ,  C12N9/22 ,  C12N15/11 ,  C12N15/111 ,  C12N15/86 ,  C12N2320/33 ,  C12N2750/14143

Abstract: Disclosed herein are engineered ADAR systems for gene editing.

公开(公告)号：US20230139474A1

公开(公告)日：2023-05-04

申请号：US17822338

申请日：2022-08-25

Applicant: The Regents of the University of California

Inventor： Prashant Mali ,  Dhruva Katrekar

IPC: C12N15/115 ,  A61P21/00 ,  C12N9/78

Abstract: Aspects of the disclosure relate to a gene therapy approach for diseases, disorders, or conditions caused by mutation in the stop codon utilizing modified tRNA. At least 10-15% of all genetic diseases, including muscular dystrophy (e.g. Duchene muscular dystrophy), some cancers, beta thalassemia, Hurler syndrome, and cystic fibrosis, fall into this category. Not to be bound by theory, it is believed that this approach is safer than CRISPR approaches due to minimal off-target effects and the lack of genome level changes.

公开(公告)号：US20230053353A1

公开(公告)日：2023-02-23

申请号：US17791498

申请日：2021-01-08

Applicant: The Regents of the University of California

Inventor： Prashant Mali ,  Dhruva Katrekar ,  Thomas Morton

IPC: C12N15/11 ,  C12N15/63 ,  C12N15/10

Abstract: The disclosure provides for a targeting transfer RNA (ttRNA) that that suppresses nonsense mutations in messenger RNA, that comprises an anticodon sequence that binds to a stop codon and a variable loop sequence that comprises an RNA aptamer that has strong binding affinity to an RNA binding protein; and methods of use thereof.

公开(公告)号：US11479775B2

公开(公告)日：2022-10-25

申请号：US16864911

申请日：2020-05-01

Applicant: The Regents of the University of California

Inventor： Prashant Mali ,  Dhruva Katrekar

IPC: C12N15/115 ,  A61P21/00 ,  C12N9/78 ,  A61K38/00 ,  C12N15/113

Abstract: Aspects of the disclosure relate to a gene therapy approach for diseases, disorders, or conditions caused by mutation in the stop codon utilizing modified tRNA. At least 10-15% of all genetic diseases, including muscular dystrophy (e.g. Duchene muscular dystrophy), some cancers, beta thalassemia, Hurler syndrome, and cystic fibrosis, fall into this category. Not to be bound by theory, it is believed that this approach is safer than CRISPR approaches due to minimal off-target effects and the lack of genome level changes.

公开(公告)号：US20220307013A1

公开(公告)日：2022-09-29

申请号：US17638428

申请日：2020-08-28

Applicant: The Regents of the University of California

Inventor： Prashant Mali ,  Kyle M. Ford ,  Nathan Palmer ,  Rebecca Panwala

IPC: C12N15/10 ,  C07K14/47 ,  C07K14/71 ,  C07K14/82

Abstract: The disclosure provides for screening methodologies using gene fragment overexpression that provide for the identification of peptide sequences which can modulate the functional regions of proteins of interests, and uses thereof. The disclosure further relates to peptide, polypeptide and polynucleotide identified by the methods of the disclosure, compositions containing such peptide, polypeptide and polynucleotides and uses thereof.

公开(公告)号：US20220202957A1

公开(公告)日：2022-06-30

申请号：US17602150

申请日：2020-04-09

Applicant: The Regents of the University of California

Inventor： Prashant Mali ,  Ana Moreno

IPC: A61K48/00 ,  A61P25/04 ,  C07K19/00 ,  C12N15/86 ,  C12N9/22

Abstract: The disclosure provides epigenetic based approaches and methods using genome editing constructs comprising a zinc finger fused with a repressor domain and/or a dCas9 fused with a repressor domain to treat and manage pain in subjects in need of treatment thereof.

公开(公告)号：US20220186226A1

公开(公告)日：2022-06-16

申请号：US17687411

申请日：2022-03-04

Applicant: The Regents of the University of California

Inventor： Prashant Mali ,  Dhruva Katrekar

IPC: C12N15/115 ,  A61P21/00 ,  C12N9/78

Abstract: Aspects of the disclosure relate to a gene therapy approach for diseases, disorders, or conditions caused by mutation in the stop codon utilizing modified tRNA. At least 10-15% of all genetic diseases, including muscular dystrophy (e.g. Duchene muscular dystrophy), some cancers, beta thalassemia, Hurler syndrome, and cystic fibrosis, fall into this category. Not to be bound by theory, it is believed that this approach is safer than CRISPR approaches due to minimal off-target effects and the lack of genome level changes.