SG11201901531TA - Regulation of gene expression using engineered nucleases - Google Patents
Regulation of gene expression using engineered nucleasesInfo
- Publication number
- SG11201901531TA SG11201901531TA SG11201901531TA SG11201901531TA SG11201901531TA SG 11201901531T A SG11201901531T A SG 11201901531TA SG 11201901531T A SG11201901531T A SG 11201901531TA SG 11201901531T A SG11201901531T A SG 11201901531TA SG 11201901531T A SG11201901531T A SG 11201901531TA
- Authority
- SG
- Singapore
- Prior art keywords
- international
- suite
- california
- richmond
- august
- Prior art date
Links
- 230000014493 regulation of gene expression Effects 0.000 title abstract 2
- 101710163270 Nuclease Proteins 0.000 title 1
- 239000003814 drug Substances 0.000 abstract 3
- CXRFDZFCGOPDTD-UHFFFAOYSA-M Cetrimide Chemical compound [Br-].CCCCCCCCCCCCCC[N+](C)(C)C CXRFDZFCGOPDTD-UHFFFAOYSA-M 0.000 abstract 2
- 239000003795 chemical substances by application Substances 0.000 abstract 2
- 238000010362 genome editing Methods 0.000 abstract 1
- 108060003196 globin Proteins 0.000 abstract 1
- 102000018146 globin Human genes 0.000 abstract 1
- 210000003958 hematopoietic stem cell Anatomy 0.000 abstract 1
- 230000004048 modification Effects 0.000 abstract 1
- 238000012986 modification Methods 0.000 abstract 1
- 230000008520 organization Effects 0.000 abstract 1
Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases RNAses, DNAses
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/06—Antianaemics
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K2035/124—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells the cells being hematopoietic, bone marrow derived or blood cells
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
- C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
Abstract
INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 01 March 2018 (01.03.2018) W I PO I PCT limionommiollmonolooliiiimilomomolioluovoimill (10) International Publication Number WO 2018/039440 Al (51) International Patent Classification: C07K 14/47 (2006.01) A61K 35/12 (2015.01) C12N 9/16 (2006.01) A61K 38/46 (2006.01) C12N 15/85 (2006.01) (21) International Application Number: PCT/US2017/048397 (22) International Filing Date: 24 August 2017 (24.08.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/378,978 24 August 2016 (24.08.2016) US 62/443,981 09 January 2017 (09.01.2017) US 62/545,778 15 August 2017 (15.08.2017) US (71) Applicant: SANGAMO THERAPEUTICS, INC. [US/US]; Point Richmond Tech Center, 501 Canal Blvd., Suite A100, Richmond, California 94804 (US). (72) Inventors: MILLER, Jeffrey C.; c/o Sangamo Therapeu- tics, Inc., Point Richmond Tech Center, 501 Canal Blvd., Suite A100, Richmond, California 94804 (US). REBAR, Edward J.; c/o Sangamo Therapeutics, Inc., Point Rich- mond Tech Center, 501 Canal Blvd., Suite A100, Rich- mond, California 94804 (US). (74) Agent: PASTERNAK, Dahna S.; Pasternak Patent Law, 1900 Embarcadero Road, Suite 211, Palo Alto, California 94303 (US). (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. (84) Designated States (unless otherwise indicated, for every kind of regional protection available): ARIPO (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, (54) Title: REGULATION OF GENE EXPRESSION USING ENGINEERED NUCLEASES 25.00 20.00 15.00 10.00 % 7 Glo bin Pro te in 0.00 U I 5.00 0 untransfected 63014/65722 untransfected 63014/65722 PB-M R-003 PB-M R-004 7/ % (Left) 7/Total 13 % (Right) FIG. 6 1-1 0 (57) : The present disclosure is in the field of genome engineering, particularly targeted modification of the genome of a \" hematopoietic cell. C [Continued on next page] WO 2018/039440 Al 1#110figinigE101010111001MOHOHOEMEEM TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). Published: with international search report (Art. 21(3)) before the expiration of the time limit for amending the claims and to be republished in the event of receipt of amendments (Rule 48.2(h))
Applications Claiming Priority (4)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201662378978P | 2016-08-24 | 2016-08-24 | |
US201762443981P | 2017-01-09 | 2017-01-09 | |
US201762545778P | 2017-08-15 | 2017-08-15 | |
PCT/US2017/048397 WO2018039440A1 (en) | 2016-08-24 | 2017-08-24 | Regulation of gene expression using engineered nucleases |
Publications (1)
Publication Number | Publication Date |
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SG11201901531TA true SG11201901531TA (en) | 2019-03-28 |
Family
ID=61246246
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG10201913315SA SG10201913315SA (en) | 2016-08-24 | 2017-08-24 | Regulation of gene expression using engineered nucleases |
SG11201901531TA SG11201901531TA (en) | 2016-08-24 | 2017-08-24 | Regulation of gene expression using engineered nucleases |
Family Applications Before (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG10201913315SA SG10201913315SA (en) | 2016-08-24 | 2017-08-24 | Regulation of gene expression using engineered nucleases |
Country Status (32)
Country | Link |
---|---|
US (2) | US10563184B2 (en) |
EP (2) | EP3504229B1 (en) |
JP (1) | JP7203014B2 (en) |
KR (1) | KR102572759B1 (en) |
CN (1) | CN110325635B (en) |
AU (2) | AU2017315406B2 (en) |
BR (1) | BR112019003100A2 (en) |
CA (1) | CA3034882A1 (en) |
CL (1) | CL2019000399A1 (en) |
CO (1) | CO2019001364A2 (en) |
CR (1) | CR20190098A (en) |
DK (1) | DK3504229T3 (en) |
DO (1) | DOP2019000031A (en) |
EC (1) | ECSP19017082A (en) |
ES (1) | ES2900317T3 (en) |
HR (1) | HRP20212025T1 (en) |
HU (1) | HUE056707T2 (en) |
IL (1) | IL264639B2 (en) |
LT (1) | LT3504229T (en) |
MX (1) | MX2019002207A (en) |
MY (1) | MY193234A (en) |
NZ (1) | NZ750938A (en) |
PE (1) | PE20190568A1 (en) |
PH (1) | PH12019500281A1 (en) |
PL (1) | PL3504229T3 (en) |
PT (1) | PT3504229T (en) |
RS (1) | RS62758B1 (en) |
SG (2) | SG10201913315SA (en) |
SI (1) | SI3504229T1 (en) |
TN (1) | TN2019000048A1 (en) |
WO (1) | WO2018039440A1 (en) |
ZA (1) | ZA201900765B (en) |
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US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
US20150166982A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting pi3k point mutations |
WO2016022363A2 (en) | 2014-07-30 | 2016-02-11 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
JP7109784B2 (en) | 2015-10-23 | 2022-08-01 | プレジデント アンド フェローズ オブ ハーバード カレッジ | Evolved Cas9 protein for gene editing |
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US11661590B2 (en) | 2016-08-09 | 2023-05-30 | President And Fellows Of Harvard College | Programmable CAS9-recombinase fusion proteins and uses thereof |
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US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
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