公开(公告)号：US20240124870A1

公开(公告)日：2024-04-18

申请号：US18332029

申请日：2023-06-09

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： Craig Cameron Mello ,  Krishna Sumanth Ghanta ,  Gregoriy Dokshin ,  Aamir Mir ,  Hassan Gneid ,  Jonathan Kenneth Watts ,  Erik Joseph Sontheimer

IPC: C12N15/10 ,  C12N9/22 ,  C12N15/113 ,  C12N15/66 ,  C12N15/90

CPC classification number: C12N15/102 ,  C12N9/22 ,  C12N15/113 ,  C12N15/66 ,  C12N15/902

Abstract: The disclosure provides novel methods and compositions for gene editing. In particular, the disclosure relates to compositions and methods of making modified nucleic acid donor templates for highly efficient and precise gene editing.

公开(公告)号：US20250034575A1

公开(公告)日：2025-01-30

申请号：US18666191

申请日：2024-05-16

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： Michael Czech ,  Batuhan Orbay Yenilmez ,  Anastasia Khvorova ,  Jonathan Kenneth Watts

IPC: C12N15/113

Abstract: This disclosure relates to the combination of DGAT2 targeting sequences with FASN, OPN, and/or NOX4 targeting sequences, and methods for treating and preventing non-alcoholic fatty liver disease (NAFLD), non-alcoholic steatohepatitis (NASH), and lipodystrophy syndromes.

公开(公告)号：US20250034548A1

公开(公告)日：2025-01-30

申请号：US18678835

申请日：2024-05-30

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： Erik Joseph Sontheimer ,  Jonathan Kenneth Watts ,  Wen Xue ,  Zexiang Chen ,  Bin Liu

IPC: C12N15/10 ,  C12N9/12 ,  C12N9/22 ,  C12N15/11

Abstract: Provided are modular prime editing systems, comprising: i) a fusion protein comprising a Cas9 nickase protein linked to a nucleotide polymerase (NT) protein, ii) a prime editor template RNA (petRNA) comprising a primer binding site (PBS), a nucleotide polymerase template (NPT), and at least one MS2 hairpin, and iii) a single guide RNA (sgRNA).

公开(公告)号：US20210388348A1

公开(公告)日：2021-12-16

申请号：US17318846

申请日：2021-05-12

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： Erik Joseph Sontheimer ,  Anastasia Khvorova ,  Jonathan Kenneth Watts ,  Nadia Amrani ,  Zexiang Chen ,  Matthew Hassler ,  Dimas Echeverria Moreno ,  Julia Frances Alterman ,  Scot Wolfe ,  Ken Yamada ,  Gitali Devi ,  Han Zhang

IPC: C12N15/11 ,  C12N15/90 ,  C12N9/22

Abstract: Chemically modified crRNAs and tracrRNAs are provided. crRNAs and tracrRNAs with 5′ and/or 3′ conjugated moieties are provided. crRNAs and tracrRNAs with modifications in the repeat region of the crRNA or the anti-repeat region of the tracrRNA are provided. Methods of using the crRNAs and tracrRNAs for genome editing with a CRISPR nuclease and kits for performing the same are also provided.

公开(公告)号：US20240309396A1

公开(公告)日：2024-09-19

申请号：US18424165

申请日：2024-01-26

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： Jonathan Kenneth Watts ,  Erik Joseph Sontheimer ,  Craig Cameron Mello ,  Zexiang Chen ,  Krishna Sumanth Ghanta

IPC: C12N15/85 ,  C12N15/11

CPC classification number: C12N15/85 ,  C12N15/11 ,  C12N2310/315 ,  C12N2310/3519

Abstract: The disclosure provides methods and compositions for gene editing. In particular. the disclosure relates to compositions and methods of making and using modified nucleic acid donor templates for highly efficient and precise gene editing, such as gen editing in immune cells.

公开(公告)号：US11981892B2

公开(公告)日：2024-05-14

申请号：US16384612

申请日：2019-04-15

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： Craig Cameron Mello ,  Krishna Sumanth Ghanta ,  Gregoriy Dokshin ,  Aamir Mir ,  Hassan Gneid ,  Jonathan Kenneth Watts ,  Erik Joseph Sontheimer

IPC: C12N15/10 ,  C12N9/22 ,  C12N15/113 ,  C12N15/66 ,  C12N15/90

CPC classification number: C12N15/102 ,  C12N9/22 ,  C12N15/113 ,  C12N15/66 ,  C12N15/902

Abstract: The disclosure provides novel methods and compositions for gene editing. In particular, the disclosure relates to compositions and methods of making modified nucleic acid donor templates for highly efficient and precise gene editing.

公开(公告)号：US20230407295A1

公开(公告)日：2023-12-21

申请号：US18141166

申请日：2023-04-28

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： Han Zhang ,  Erik Joseph Sontheimer ,  Jonathan Kenneth Watts ,  Anastasia Khvorova

IPC: C12N15/11 ,  C12N9/22 ,  C12N15/90

CPC classification number: C12N15/11 ,  C12N9/22 ,  C12N15/907 ,  C12N2310/322 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/20

Abstract: Protecting oligonucleotides are provided. Protecting oligonucleotides with 5′ and/or 3′ conjugated moieties are provided. Protecting oligonucleotides with chemical modifications are provided. Methods of using the protecting oligonucleotides for genome editing with a CRISPR nuclease and kits for performing the same are also provided.

公开(公告)号：US20230183687A1

公开(公告)日：2023-06-15

申请号：US17964611

申请日：2022-10-12

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： Erik Joseph Sontheimer ,  Jonathan Kenneth Watts ,  Anastasia Khvorova ,  Nathan Bamidele

IPC: C12N15/11 ,  C12N9/22 ,  C12N15/90

CPC classification number: C12N15/11 ,  C12N15/111 ,  C12N9/22 ,  C12N15/907 ,  C12N2310/20 ,  C12N2800/80 ,  C12N2310/314

Abstract: Chemically modified Neisseria meningitidis (Nme) crRNAs, tracrRNAs, and sgRNAs are provided. Methods of using the Nme crRNAs, tracrRNAs, and sgRNAs for genome editing with a Nme CRISPR nuclease and kits for performing the same are also provided.