Title:
NUCLEIC ACID EDITING SYSTEMS, METHODS, AND USES THEREOF
Document Type and Number:
WIPO Patent Application WO/2023/173062
Kind Code:
A3
Abstract:
Nucleic acid editing systems, methods, and uses thereof are provided. Various nuclease amino acid sequences and guide RNA sequences are described that promote cleavage of a target nucleic acid sequence. Methods of treating a disease and methods of detecting an alternation in a gene are described. Scaffolds and fusion proteins comprising nuclease mutants and fragments thereof are also provided.
Inventors:
BUERCKSTUEMMER TILMANN (AT)
SCHMIDT HENRIK (AT)
SIEBER ANNA (AT)
ILSINGER CHRISTOPH (AT)
SCHMIDT HENRIK (AT)
SIEBER ANNA (AT)
ILSINGER CHRISTOPH (AT)
Application Number:
PCT/US2023/064097
Publication Date:
November 23, 2023
Filing Date:
March 10, 2023
Export Citation:
Assignee:
INTIMA BIOSCIENCE INC (US)
International Classes:
C12N15/11; C12N9/22; C12N15/10; C12N15/113; C12N15/63
Foreign References:
| US11098325B2 | 2021-08-24 | |||
| US20180237770A1 | 2018-08-23 | |||
| US20180245099A1 | 2018-08-30 | |||
| US20200263190A1 | 2020-08-20 | |||
| US20210284979A1 | 2021-09-16 |
Other References:
DATABASE Protein 13 October 2019 (2019-10-13), ANONYMOUS: "type V CRISPR-associated protein Cas12a/Cpf1 [Xylanibacter ruminicola]", XP093113549, retrieved from Genbank Database accession no. WP_073043853
S.-T. CHOU, Q. LENG, J. MIXSON: "Zinc finger nucleases: Tailor-made for gene therapy", DRUGS OF THE FUTURE, vol. 37, no. 3, 1 January 2012 (2012-01-01), ES , pages 183 - 196, XP055512500, ISSN: 0377-8282, DOI: 10.1358/dof.2012.037.03.1779022
BĄBAŁA NIKOLINA, BOVENS ASTRID, DE VRIES EVERT, IGLESIAS-GUIMARAIS VICTORIA, AHRENDS TOMASZ, KRUMMEL MATTHEW F., BORST JANNIE, BIN: "Subcellular Localization of Antigen in Keratinocytes Dictates Delivery of CD4+ T-cell Help for the CTL Response upon Therapeutic DNA Vaccination into the Skin", CANCER IMMUNOLOGY RESEARCH, vol. 6, no. 7, 1 July 2018 (2018-07-01), US , pages 835 - 847, XP093113555, ISSN: 2326-6066, DOI: 10.1158/2326-6066.CIR-17-0408
DATABASE Nucleotide 31 May 2016 (2016-05-31), ANONYMOUS: "PREDICTED: Anolis carolinensis EF-hand calcium-binding domain-containing protein 2-like(LOC100563576), mRNA", XP093113556, retrieved from Genbank Database accession no. XM_003230113
DATABASE Protein 22 April 2021 (2021-04-22), ANONYMOUS: "MAG: type V CRISPR-associated protein Cas12a/Cpf1 [Prevotella sp.", XP093113564, retrieved from Genbank Database accession no. MBR3078291
S.-T. CHOU, Q. LENG, J. MIXSON: "Zinc finger nucleases: Tailor-made for gene therapy", DRUGS OF THE FUTURE, vol. 37, no. 3, 1 January 2012 (2012-01-01), ES , pages 183 - 196, XP055512500, ISSN: 0377-8282, DOI: 10.1358/dof.2012.037.03.1779022
BĄBAŁA NIKOLINA, BOVENS ASTRID, DE VRIES EVERT, IGLESIAS-GUIMARAIS VICTORIA, AHRENDS TOMASZ, KRUMMEL MATTHEW F., BORST JANNIE, BIN: "Subcellular Localization of Antigen in Keratinocytes Dictates Delivery of CD4+ T-cell Help for the CTL Response upon Therapeutic DNA Vaccination into the Skin", CANCER IMMUNOLOGY RESEARCH, vol. 6, no. 7, 1 July 2018 (2018-07-01), US , pages 835 - 847, XP093113555, ISSN: 2326-6066, DOI: 10.1158/2326-6066.CIR-17-0408
DATABASE Nucleotide 31 May 2016 (2016-05-31), ANONYMOUS: "PREDICTED: Anolis carolinensis EF-hand calcium-binding domain-containing protein 2-like(LOC100563576), mRNA", XP093113556, retrieved from Genbank Database accession no. XM_003230113
DATABASE Protein 22 April 2021 (2021-04-22), ANONYMOUS: "MAG: type V CRISPR-associated protein Cas12a/Cpf1 [Prevotella sp.", XP093113564, retrieved from Genbank Database accession no. MBR3078291
Attorney, Agent or Firm:
ANDERSON, Stephen G. et al. (US)
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