HRP20212025T1 - Regulacija ekspresije gena pomoću projektiranih nukleaza - Google Patents
Regulacija ekspresije gena pomoću projektiranih nukleaza Download PDFInfo
- Publication number
- HRP20212025T1 HRP20212025T1 HRP20212025TT HRP20212025T HRP20212025T1 HR P20212025 T1 HRP20212025 T1 HR P20212025T1 HR P20212025T T HRP20212025T T HR P20212025TT HR P20212025 T HRP20212025 T HR P20212025T HR P20212025 T1 HRP20212025 T1 HR P20212025T1
- Authority
- HR
- Croatia
- Prior art keywords
- cell
- zinc finger
- polynucleotides
- finger nuclease
- sequence
- Prior art date
Links
- 101710163270 Nuclease Proteins 0.000 title 1
- 230000014493 regulation of gene expression Effects 0.000 title 1
- 210000004027 cell Anatomy 0.000 claims 45
- 108010017070 Zinc Finger Nucleases Proteins 0.000 claims 13
- 108091033319 polynucleotide Proteins 0.000 claims 11
- 102000040430 polynucleotide Human genes 0.000 claims 11
- 239000002157 polynucleotide Substances 0.000 claims 11
- 238000000034 method Methods 0.000 claims 10
- 102100022976 B-cell lymphoma/leukemia 11A Human genes 0.000 claims 7
- 101000903703 Homo sapiens B-cell lymphoma/leukemia 11A Proteins 0.000 claims 7
- 239000013598 vector Substances 0.000 claims 6
- 239000003623 enhancer Substances 0.000 claims 4
- 238000010362 genome editing Methods 0.000 claims 4
- 239000000825 pharmaceutical preparation Substances 0.000 claims 4
- 108700028146 Genetic Enhancer Elements Proteins 0.000 claims 3
- 210000000130 stem cell Anatomy 0.000 claims 3
- 208000002903 Thalassemia Diseases 0.000 claims 2
- 125000003275 alpha amino acid group Chemical group 0.000 claims 2
- 208000005980 beta thalassemia Diseases 0.000 claims 2
- 238000012217 deletion Methods 0.000 claims 2
- 230000037430 deletion Effects 0.000 claims 2
- 108020001507 fusion proteins Proteins 0.000 claims 2
- 102000037865 fusion proteins Human genes 0.000 claims 2
- 208000034737 hemoglobinopathy Diseases 0.000 claims 2
- 238000000338 in vitro Methods 0.000 claims 2
- 230000001400 myeloablative effect Effects 0.000 claims 2
- 208000007056 sickle cell anemia Diseases 0.000 claims 2
- 102000004127 Cytokines Human genes 0.000 claims 1
- 108090000695 Cytokines Proteins 0.000 claims 1
- 102100021519 Hemoglobin subunit beta Human genes 0.000 claims 1
- 108091005904 Hemoglobin subunit beta Proteins 0.000 claims 1
- 108091005886 Hemoglobin subunit gamma Proteins 0.000 claims 1
- 102100038617 Hemoglobin subunit gamma-2 Human genes 0.000 claims 1
- 210000001185 bone marrow Anatomy 0.000 claims 1
- 230000003750 conditioning effect Effects 0.000 claims 1
- 210000005260 human cell Anatomy 0.000 claims 1
- 238000001727 in vivo Methods 0.000 claims 1
- 238000003780 insertion Methods 0.000 claims 1
- 230000037431 insertion Effects 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 108020004999 messenger RNA Proteins 0.000 claims 1
- 238000012986 modification Methods 0.000 claims 1
- 230000004048 modification Effects 0.000 claims 1
- 239000002243 precursor Substances 0.000 claims 1
- 230000000644 propagated effect Effects 0.000 claims 1
- 230000004936 stimulating effect Effects 0.000 claims 1
Classifications
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Cell Biology (AREA)
- Hematology (AREA)
- Immunology (AREA)
- Developmental Biology & Embryology (AREA)
- Mycology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Diabetes (AREA)
- Epidemiology (AREA)
- Virology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
- Enzymes And Modification Thereof (AREA)
Claims (17)
1. Nukleaza cinkovog prsta (ZFN) koja sadrži aminokiselinsku sekvencu kako je prikazano u SEQ ID NO:29 i aminokiselinsku sekvencu kao što je prikazano u SEQ ID NO:31.
2. Jedan ili više polinukleotida koji kodiraju jednu ili više nukleaza cinkovog prsta prema patentnom zahtjevu 1, po izboru pri čemu jedan ili više polinukleotida je (su) mRNA.
3. Jedan ili više polinukleotida prema patentnom zahtjevu 2, naznačen time što sadrži SEQ ID NO:28 i SEQ ID NO:30.
4. Jedan ili više vektora naznačeni time što sadrže sekvence koje kodiraju jednu ili više nukleaza cinkovog prsta prema zahtjevu 1.
5. Stanica naznačena time što sadrži nukleazu cinkovog prsta prema zahtjevu 1, jedan ili više polinukleotida prema zahtjevu 2 ili zahtjevu 3 ili jedan ili više vektora prema zahtjevu 4.
6. Stanica prema patentnom zahtjevu 5, naznačena time što:
(i) stanica je matična stanica ili prekursorska stanica, po izboru pri čemu je stanica ljudska stanica; i/ili
(ii) genom stanice je modificiran nukleazom cinkovog prsta, po izboru pri čemu je genomska modifikacija odabrana iz skupine koja se sastoji od insercija, delecija i njihovih kombinacija, kao što je genomska modifikacija unutar +58 regije sekvence pojačivača BCL11A.
7. Stanica ili stanična linija proizvedena iz stanice prema patentnom zahtjevu 5 ili patentnom zahtjevu 6, naznačena time što sadrži nukleazu cinkovog prsta prema patentnom zahtjevu 1, jedan ili više polinukleotida prema patentnom zahtjevu 2 ili patentnom zahtjevu 3 ili jedan ili više vektora prema patentnom zahtjevu 4.
8. Djelomično ili potpuno diferencirana stanica koja potječe od stanice ili stanične linije prema bilo kojem od patentnih zahtjeva 5 do 7, naznačena time što sadrži nukleazu cinkovog prsta prema patentnom zahtjevu 1, jedan ili više polinukleotida prema patentnom zahtjevu 2 ili patentnom zahtjevu 3 ili jedan ili više vektora prema patentnom zahtjevu 4.
9. Stanica ili stanična linija prema bilo kojem od patentnih zahtjeva 5 do 8, naznačena time što je genom stanice modificiran nukleazom cinkovog prsta i stanica ili stanična linija pokazuju povećanu ekspresiju gama i/ili beta globina u usporedbi sa stanicom ili staničnom linijom bez genomske modifikacije.
10. Farmaceutski pripravak naznačen time što sadrži nukleazu cinkovog prsta prema patentnom zahtjevu 1, jedan ili više polinukleotida prema patentnom zahtjevu 2 ili patentnom zahtjevu 3, jedan ili više vektora prema patentnom zahtjevu 4, i/ili stanicu ili staničnu liniju prema bilo kojem od patentnih zahtjeva 5-9.
11. Farmaceutski pripravak prema zahtjevu 10, naznačen time što sadrži stanicu ili staničnu liniju prema bilo kojem od patentnih zahtjeva 5-9 i nadalje sadrži propagirane i diferencirane stanice koje potječu od stanice ili stanične linije.
12. In vitro postupak za modificiranje endogene sekvence pojačivača BCL11A u stanici, te postupak obuhvaća davanje nukleaze cinkovog prsta prema patentnom zahtjevu 1 ili jednog ili više polinukleotida prema patentnom zahtjevu 2 ili patentnom zahtjevu 3 u stanicu tako da je izmijenjena endogena sekvence pojačivača BCL11A, po izboru pri čemu:
(i) postupak dalje uključuje uvođenje egzogene sekvence u stanicu tako da se egzogena sekvenca umetne u endogenu sekvencu pojačivača BCL11A; ili
(ii) modifikacija uključuje brisanje.
13. Farmaceutski pripravak prema patentnom zahtjevu 10 ili patentnom zahtjevu 11 za upotrebu u postupku liječenja hemoglobinopatije, kao što je talasemija (npr. beta-talasemija) ili bolest srpastih stanica, kod pacijenta, te se postupak sastoji od davanja farmaceutskog pripravka pacijentu, izborno pri čemu je pacijent čovjek.
14. In vitro postupak za proizvodnju genetski modificirane stanice koja sadrži genomsku modifikaciju unutar endogene sekvence pojačivača BCL11A, te postupak obuhvaća korake:
a) dovođenje u kontakt stanice s jednim ili više polinukleotida prema zahtjevu 2 ili zahtjevu 3;
b) podvrgavanje stanice uvjetima pogodnim za ekspresiju fuzijskog proteina iz polinukleotida; i
c) modificiranje endogene sekvence pojačivača BCL11A s eksprimiranim fuzijskim proteinom dovoljnim za proizvodnju genetski modificirane stanice,
po izboru pri čemu postupak dalje uključuje stimulaciju stanice s najmanje jednim citokinom.
15. Komplet naznačen time što sadrži nukleazu cinkovog prsta prema patentnom zahtjevu 1, jedan ili više polinukleotida prema patentnom zahtjevu 2 ili patentnom zahtjevu 3, jedan ili više vektora prema patentnom zahtjevu 4 i/ili stanicu ili staničnu liniju prema bilo kojem od patentnih zahtjeva 5-9.
16. Stanica ili stanična linija prema bilo kojem od patentnih zahtjeva 5-9 za upotrebu u postupku liječenja ili prevencije hemoglobinopatije, kao što je talasemija (npr. beta-talasemija) ili bolest srpastih stanica.
17. Stanica ili stanična linija za uporabu u skladu s patentnim zahtjevom 16, naznačena time što je stanica ljudska matična stanica ili progenitorska stanica i genom stanice je modificiran unutar sekvence pojačivača BCL11A pomoću nukleaze cinkovog prsta, po izboru pri čemu:
(i) stanice su ekspandirane prije davanja subjektu;
(ii) stanice se daju subjektu u transplantaciji koštane srži, gdje se stanice ugrađuju, diferenciraju i sazrijevaju in vivo; i/ili
(iii) prije uvođenja transplantata koji sadrži stanice, subjekt se liječi blagim mijeloablativnim postupkom ili snažnim mijeloablativnim režimom kondicioniranja.
Applications Claiming Priority (5)
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 | |
EP17844404.8A EP3504229B1 (en) | 2016-08-24 | 2017-08-24 | Regulation of gene expression using engineered nucleases |
PCT/US2017/048397 WO2018039440A1 (en) | 2016-08-24 | 2017-08-24 | Regulation of gene expression using engineered nucleases |
Publications (1)
Publication Number | Publication Date |
---|---|
HRP20212025T1 true HRP20212025T1 (hr) | 2022-04-01 |
Family
ID=61246246
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
HRP20212025TT HRP20212025T1 (hr) | 2016-08-24 | 2017-08-24 | Regulacija ekspresije gena pomoću projektiranih nukleaza |
Country Status (32)
Country | Link |
---|---|
US (2) | US10563184B2 (hr) |
EP (2) | EP3504229B1 (hr) |
JP (1) | JP7203014B2 (hr) |
KR (1) | KR102572759B1 (hr) |
CN (1) | CN110325635B (hr) |
AU (2) | AU2017315406B2 (hr) |
BR (1) | BR112019003100A2 (hr) |
CA (1) | CA3034882A1 (hr) |
CL (1) | CL2019000399A1 (hr) |
CO (1) | CO2019001364A2 (hr) |
CR (1) | CR20190098A (hr) |
DK (1) | DK3504229T3 (hr) |
DO (1) | DOP2019000031A (hr) |
EC (1) | ECSP19017082A (hr) |
ES (1) | ES2900317T3 (hr) |
HR (1) | HRP20212025T1 (hr) |
HU (1) | HUE056707T2 (hr) |
IL (1) | IL264639B2 (hr) |
LT (1) | LT3504229T (hr) |
MX (1) | MX2019002207A (hr) |
MY (1) | MY193234A (hr) |
NZ (1) | NZ750938A (hr) |
PE (1) | PE20190568A1 (hr) |
PH (1) | PH12019500281A1 (hr) |
PL (1) | PL3504229T3 (hr) |
PT (1) | PT3504229T (hr) |
RS (1) | RS62758B1 (hr) |
SG (2) | SG10201913315SA (hr) |
SI (1) | SI3504229T1 (hr) |
TN (1) | TN2019000048A1 (hr) |
WO (1) | WO2018039440A1 (hr) |
ZA (1) | ZA201900765B (hr) |
Families Citing this family (32)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP3613852A3 (en) | 2011-07-22 | 2020-04-22 | President and Fellows of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
US9163284B2 (en) | 2013-08-09 | 2015-10-20 | President And Fellows Of Harvard College | Methods for identifying a target site of a Cas9 nuclease |
US9359599B2 (en) | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
US20150166982A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting pi3k point mutations |
US10077453B2 (en) | 2014-07-30 | 2018-09-18 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
IL294014B1 (en) | 2015-10-23 | 2024-03-01 | Harvard College | Nucleobase editors and their uses |
AU2017306676B2 (en) | 2016-08-03 | 2024-02-22 | President And Fellows Of Harvard College | Adenosine nucleobase editors and uses thereof |
CA3033327A1 (en) | 2016-08-09 | 2018-02-15 | President And Fellows Of Harvard College | Programmable cas9-recombinase fusion proteins and uses thereof |
WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
PL3504229T3 (pl) * | 2016-08-24 | 2022-04-19 | Sangamo Therapeutics, Inc. | Regulacja ekspresji genów przy użyciu nukleaz poddanych inżynierii |
EP3504327B1 (en) | 2016-08-24 | 2021-10-06 | Sangamo Therapeutics, Inc. | Engineered target specific nucleases |
CN110214180A (zh) | 2016-10-14 | 2019-09-06 | 哈佛大学的校长及成员们 | 核碱基编辑器的aav递送 |
EP3548616A4 (en) * | 2016-12-01 | 2020-05-13 | Sangamo Therapeutics, Inc. | TAU MODULATORS AND METHODS AND COMPOSITIONS FOR THEIR ADMINISTRATION |
WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
TW201839136A (zh) | 2017-02-06 | 2018-11-01 | 瑞士商諾華公司 | 治療血色素異常症之組合物及方法 |
US11898179B2 (en) | 2017-03-09 | 2024-02-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
JP2020510439A (ja) | 2017-03-10 | 2020-04-09 | 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 | シトシンからグアニンへの塩基編集因子 |
KR20190130613A (ko) | 2017-03-23 | 2019-11-22 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제 |
WO2018209320A1 (en) | 2017-05-12 | 2018-11-15 | President And Fellows Of Harvard College | Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation |
WO2019023680A1 (en) | 2017-07-28 | 2019-01-31 | President And Fellows Of Harvard College | METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE) |
US11319532B2 (en) | 2017-08-30 | 2022-05-03 | President And Fellows Of Harvard College | High efficiency base editors comprising Gam |
US11795443B2 (en) | 2017-10-16 | 2023-10-24 | The Broad Institute, Inc. | Uses of adenosine base editors |
CA3089587A1 (en) | 2018-02-08 | 2019-08-15 | Sangamo Therapeutics, Inc. | Engineered target specific nucleases |
EP3942040A1 (en) | 2019-03-19 | 2022-01-26 | The Broad Institute, Inc. | Methods and compositions for editing nucleotide sequences |
MX2021012152A (es) | 2019-04-02 | 2021-11-03 | Sangamo Therapeutics Inc | Metodos para el tratamiento de beta-talasemia. |
MX2022014008A (es) | 2020-05-08 | 2023-02-09 | Broad Inst Inc | Métodos y composiciones para la edición simultánea de ambas cadenas de una secuencia de nucleótidos de doble cadena objetivo. |
CN114149990A (zh) * | 2020-09-08 | 2022-03-08 | 甘李药业股份有限公司 | 编辑造血干/祖细胞中bcl11a基因的方法 |
US11514747B2 (en) | 2020-09-30 | 2022-11-29 | Aristocrat Technologies, Inc. | Latching assembly for a pushbutton of an electronic gaming machine |
WO2023014839A1 (en) * | 2021-08-03 | 2023-02-09 | Sangamo Therapeutics, Inc. | Methods for the treatment of sickle cell disease |
Family Cites Families (123)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US4217344A (en) | 1976-06-23 | 1980-08-12 | L'oreal | Compositions containing aqueous dispersions of lipid spheres |
US4235871A (en) | 1978-02-24 | 1980-11-25 | Papahadjopoulos Demetrios P | Method of encapsulating biologically active materials in lipid vesicles |
US4186183A (en) | 1978-03-29 | 1980-01-29 | The United States Of America As Represented By The Secretary Of The Army | Liposome carriers in chemotherapy of leishmaniasis |
US4261975A (en) | 1979-09-19 | 1981-04-14 | Merck & Co., Inc. | Viral liposome particle |
US4485054A (en) | 1982-10-04 | 1984-11-27 | Lipoderm Pharmaceuticals Limited | Method of encapsulating biologically active materials in multilamellar lipid vesicles (MLV) |
US4501728A (en) | 1983-01-06 | 1985-02-26 | Technology Unlimited, Inc. | Masking of liposomes from RES recognition |
US5049386A (en) | 1985-01-07 | 1991-09-17 | Syntex (U.S.A.) Inc. | N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
US4897355A (en) | 1985-01-07 | 1990-01-30 | Syntex (U.S.A.) Inc. | N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
US4946787A (en) | 1985-01-07 | 1990-08-07 | Syntex (U.S.A.) Inc. | N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
US4797368A (en) | 1985-03-15 | 1989-01-10 | The United States Of America As Represented By The Department Of Health And Human Services | Adeno-associated virus as eukaryotic expression vector |
US4774085A (en) | 1985-07-09 | 1988-09-27 | 501 Board of Regents, Univ. of Texas | Pharmaceutical administration systems containing a mixture of immunomodulators |
US5422251A (en) | 1986-11-26 | 1995-06-06 | Princeton University | Triple-stranded nucleic acids |
US4837028A (en) | 1986-12-24 | 1989-06-06 | Liposome Technology, Inc. | Liposomes with enhanced circulation time |
US5176996A (en) | 1988-12-20 | 1993-01-05 | Baylor College Of Medicine | Method for making synthetic oligonucleotides which bind specifically to target sites on duplex DNA molecules, by forming a colinear triplex, the synthetic oligonucleotides and methods of use |
US5264618A (en) | 1990-04-19 | 1993-11-23 | Vical, Inc. | Cationic lipids for intracellular delivery of biologically active molecules |
WO1991017424A1 (en) | 1990-05-03 | 1991-11-14 | Vical, Inc. | Intracellular delivery of biologically active substances by means of self-assembling lipid complexes |
US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
US5436150A (en) | 1992-04-03 | 1995-07-25 | The Johns Hopkins University | Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease |
US5487994A (en) | 1992-04-03 | 1996-01-30 | The Johns Hopkins University | Insertion and deletion mutants of FokI restriction endonuclease |
US5356802A (en) | 1992-04-03 | 1994-10-18 | The Johns Hopkins University | Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease |
US5587308A (en) | 1992-06-02 | 1996-12-24 | The United States Of America As Represented By The Department Of Health & Human Services | Modified adeno-associated virus vector capable of expression from a novel promoter |
US6140466A (en) | 1994-01-18 | 2000-10-31 | The Scripps Research Institute | Zinc finger protein derivatives and methods therefor |
US6242568B1 (en) | 1994-01-18 | 2001-06-05 | The Scripps Research Institute | Zinc finger protein derivatives and methods therefor |
CA2681922C (en) | 1994-01-18 | 2012-05-15 | The Scripps Research Institute | Zinc finger protein derivatives and methods therefor |
EP0752005B1 (en) | 1994-03-23 | 2008-10-08 | Ohio University | Compacted nucleic acids and their delivery to cells |
GB9824544D0 (en) | 1998-11-09 | 1999-01-06 | Medical Res Council | Screening system |
JP4118327B2 (ja) | 1994-08-20 | 2008-07-16 | ゲンダック・リミテッド | Dna認識のための結合タンパク質におけるまたはそれに関連する改良 |
US5789538A (en) | 1995-02-03 | 1998-08-04 | Massachusetts Institute Of Technology | Zinc finger proteins with high affinity new DNA binding specificities |
US5925523A (en) | 1996-08-23 | 1999-07-20 | President & Fellows Of Harvard College | Intraction trap assay, reagents and uses thereof |
GB2338237B (en) | 1997-02-18 | 2001-02-28 | Actinova Ltd | In vitro peptide or protein expression library |
GB9703369D0 (en) | 1997-02-18 | 1997-04-09 | Lindqvist Bjorn H | Process |
GB9710807D0 (en) | 1997-05-23 | 1997-07-23 | Medical Res Council | Nucleic acid binding proteins |
GB9710809D0 (en) | 1997-05-23 | 1997-07-23 | Medical Res Council | Nucleic acid binding proteins |
US6995006B2 (en) | 1997-09-05 | 2006-02-07 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
US6410248B1 (en) | 1998-01-30 | 2002-06-25 | Massachusetts Institute Of Technology | General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites |
JP4309051B2 (ja) | 1998-03-02 | 2009-08-05 | マサチューセッツ インスティテュート オブ テクノロジー | 改善したリンカーを有するポリジンクフィンガータンパク質 |
US6140081A (en) | 1998-10-16 | 2000-10-31 | The Scripps Research Institute | Zinc finger binding domains for GNN |
US7070934B2 (en) | 1999-01-12 | 2006-07-04 | Sangamo Biosciences, Inc. | Ligand-controlled regulation of endogenous gene expression |
US7013219B2 (en) | 1999-01-12 | 2006-03-14 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
US6599692B1 (en) | 1999-09-14 | 2003-07-29 | Sangamo Bioscience, Inc. | Functional genomics using zinc finger proteins |
US6453242B1 (en) | 1999-01-12 | 2002-09-17 | Sangamo Biosciences, Inc. | Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites |
US6534261B1 (en) | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
US6794136B1 (en) | 2000-11-20 | 2004-09-21 | Sangamo Biosciences, Inc. | Iterative optimization in the design of binding proteins |
US7030215B2 (en) | 1999-03-24 | 2006-04-18 | Sangamo Biosciences, Inc. | Position dependent recognition of GNN nucleotide triplets by zinc fingers |
ATE309536T1 (de) | 1999-12-06 | 2005-11-15 | Sangamo Biosciences Inc | Methoden zur verwendung von randomisierten zinkfingerprotein-bibliotheken zur identifizierung von genfunktionen |
WO2001059450A2 (en) | 2000-02-08 | 2001-08-16 | Sangamo Biosciences, Inc. | Cells expressing zinc finger protein for drug discovery |
US20020061512A1 (en) | 2000-02-18 | 2002-05-23 | Kim Jin-Soo | Zinc finger domains and methods of identifying same |
AU2001263155A1 (en) | 2000-05-16 | 2001-11-26 | Massachusetts Institute Of Technology | Methods and compositions for interaction trap assays |
JP2002060786A (ja) | 2000-08-23 | 2002-02-26 | Kao Corp | 硬質表面用殺菌防汚剤 |
US7067317B2 (en) | 2000-12-07 | 2006-06-27 | Sangamo Biosciences, Inc. | Regulation of angiogenesis with zinc finger proteins |
CA2435394C (en) | 2001-01-22 | 2018-01-09 | Sangamo Biosciences, Inc. | Modified zinc finger binding proteins |
GB0108491D0 (en) | 2001-04-04 | 2001-05-23 | Gendaq Ltd | Engineering zinc fingers |
US20040224385A1 (en) | 2001-08-20 | 2004-11-11 | Barbas Carlos F | Zinc finger binding domains for cnn |
US7271002B2 (en) | 2001-11-09 | 2007-09-18 | United States Of America, Represented By The Secretary, Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
US7262054B2 (en) | 2002-01-22 | 2007-08-28 | Sangamo Biosciences, Inc. | Zinc finger proteins for DNA binding and gene regulation in plants |
AU2003251286B2 (en) | 2002-01-23 | 2007-08-16 | The University Of Utah Research Foundation | Targeted chromosomal mutagenesis using zinc finger nucleases |
US20030232410A1 (en) | 2002-03-21 | 2003-12-18 | Monika Liljedahl | Methods and compositions for using zinc finger endonucleases to enhance homologous recombination |
US7074596B2 (en) | 2002-03-25 | 2006-07-11 | Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College | Synthesis and use of anti-reverse mRNA cap analogues |
US7419817B2 (en) | 2002-05-17 | 2008-09-02 | The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. | Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography |
US7361635B2 (en) | 2002-08-29 | 2008-04-22 | Sangamo Biosciences, Inc. | Simultaneous modulation of multiple genes |
CA2497913C (en) | 2002-09-05 | 2014-06-03 | California Institute Of Technology | Use of chimeric nucleases to stimulate gene targeting |
US8409861B2 (en) | 2003-08-08 | 2013-04-02 | Sangamo Biosciences, Inc. | Targeted deletion of cellular DNA sequences |
US7888121B2 (en) | 2003-08-08 | 2011-02-15 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
US7972854B2 (en) | 2004-02-05 | 2011-07-05 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
EP1737498A2 (en) | 2004-04-08 | 2007-01-03 | Sangamo Biosciences Inc. | Zinc finger proteins for treatment of neuropathic pain |
WO2005099771A2 (en) | 2004-04-08 | 2005-10-27 | Sangamo Biosciences, Inc. | Methods and compositions for treating neuropathic and neurodegenerative conditions |
US20080131962A1 (en) | 2006-05-25 | 2008-06-05 | Sangamo Biosciences, Inc. | Engineered cleavage half-domains |
CA2579677A1 (en) | 2004-09-16 | 2006-03-30 | Sangamo Biosciences, Inc. | Compositions and methods for protein production |
EP1877583A2 (en) | 2005-05-05 | 2008-01-16 | Arizona Board of Regents on behalf of the Unversity of Arizona | Sequence enabled reassembly (seer) - a novel method for visualizing specific dna sequences |
US7951925B2 (en) | 2006-05-25 | 2011-05-31 | Sangamo Biosciences, Inc. | Methods and compositions for gene inactivation |
CN105296527B (zh) | 2006-08-11 | 2020-11-27 | 陶氏益农公司 | 锌指核酸酶介导的同源重组 |
WO2008133938A2 (en) | 2007-04-26 | 2008-11-06 | Sangamo Biosciences, Inc. | Targeted integration into the ppp1r12c locus |
US8585526B2 (en) | 2007-06-18 | 2013-11-19 | American Axle & Manufacturing, Inc. | Vehicle driveline component having heat sink for increased heat rejection capabilities |
ES2500515T3 (es) | 2007-06-19 | 2014-09-30 | Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College | Síntesis y utilización de análogos de fosforotioato antiinversos de la caperuza de ARN mensajero |
WO2009042163A2 (en) | 2007-09-27 | 2009-04-02 | Sangamo Biosciences, Inc. | Rapid in vivo identification of biologically active nucleases |
AU2008317354B2 (en) | 2007-10-25 | 2014-04-10 | Ospedale San Raffaele S.R.L. | Methods and compositions for targeted integration |
DE102007056956B4 (de) | 2007-11-27 | 2009-10-29 | Moosbauer, Peter, Dipl.-Ing.(FH) | Schaltung zur Regelung der Stromversorgung eines Verbrauchers und Verfahren zum Betrieb einer Schaltung |
EP2281050B1 (en) | 2008-04-14 | 2014-04-02 | Sangamo BioSciences, Inc. | Linear donor constructs for targeted integration |
JP2011521643A (ja) | 2008-05-28 | 2011-07-28 | サンガモ バイオサイエンシーズ, インコーポレイテッド | Dna結合ドメインおよび切断ドメインを連結するための組成物 |
KR101759586B1 (ko) | 2008-08-22 | 2017-07-19 | 상가모 테라퓨틱스, 인코포레이티드 | 표적화된 단일가닥 분할 및 표적화된 통합을 위한 방법 및 조성물 |
PT2334794T (pt) | 2008-09-15 | 2017-02-15 | Harvard College | Modulação de bcl11a para tratamento de hemoglobinopatias |
KR101803737B1 (ko) | 2008-12-04 | 2017-12-01 | 상가모 테라퓨틱스, 인코포레이티드 | 징크 핑거 뉴클레아제를 이용한 랫트의 유전체 편집 |
WO2011012316A2 (de) | 2009-07-31 | 2011-02-03 | Ludwig-Maximilians-Universität | Rna mit einer kombination aus unmodifizierten und modifizierten nucleotiden zur proteinexpression |
US8956828B2 (en) | 2009-11-10 | 2015-02-17 | Sangamo Biosciences, Inc. | Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases |
CN102762726A (zh) | 2009-11-27 | 2012-10-31 | 巴斯夫植物科学有限公司 | 嵌合内切核酸酶及其用途 |
EP2529020B1 (en) | 2010-01-28 | 2018-04-25 | The Children's Hospital of Philadelphia | A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy |
JP6137596B2 (ja) | 2010-02-08 | 2017-05-31 | サンガモ セラピューティクス, インコーポレイテッド | 遺伝子操作された切断ハーフドメイン |
EP2660318A1 (en) | 2010-02-09 | 2013-11-06 | Sangamo BioSciences, Inc. | Targeted genomic modification with partially single-stranded donor molecules |
WO2011130624A2 (en) * | 2010-04-16 | 2011-10-20 | Immune Disease Institute, Inc. | Sustained polypeptide expression from synthetic, modified rnas and uses thereof |
CN102939377B (zh) | 2010-04-26 | 2016-06-08 | 桑格摩生物科学股份有限公司 | 使用锌指核酸酶对Rosa位点进行基因组编辑 |
WO2011139349A1 (en) | 2010-05-03 | 2011-11-10 | Sangamo Biosciences, Inc. | Compositions for linking zinc finger modules |
AU2011256838B2 (en) | 2010-05-17 | 2014-10-09 | Sangamo Therapeutics, Inc. | Novel DNA-binding proteins and uses thereof |
CA2993567C (en) | 2010-07-21 | 2022-06-28 | Sangamo Biosciences, Inc. | Methods and compositions for modification of a t-cell receptor gene |
US9057057B2 (en) | 2010-07-27 | 2015-06-16 | The Johns Hopkins University | Obligate heterodimer variants of foki cleavage domain |
GB201014169D0 (en) * | 2010-08-25 | 2010-10-06 | Cambridge Entpr Ltd | In vitro hepatic differentiation |
US9405700B2 (en) | 2010-11-04 | 2016-08-02 | Sonics, Inc. | Methods and apparatus for virtualization in an integrated circuit |
CN102358902B (zh) | 2011-04-02 | 2013-01-02 | 西南大学 | 家蚕丝素重链基因突变序列及突变的方法和应用 |
EP3498833B1 (en) | 2011-09-21 | 2023-08-16 | Sangamo Therapeutics, Inc. | Methods and compositions for regulation of transgene expression |
EP2771457B1 (en) | 2011-10-27 | 2017-11-22 | Sangamo Therapeutics, Inc. | Methods and compositions for modification of the hprt locus |
US9458205B2 (en) | 2011-11-16 | 2016-10-04 | Sangamo Biosciences, Inc. | Modified DNA-binding proteins and uses thereof |
CN103361328B (zh) * | 2012-03-30 | 2017-08-18 | 复旦大学 | 介导整合的hiv‑1前病毒基因敲除的锌指核酸内切酶及其制法和应用 |
CN104471067B (zh) | 2012-05-07 | 2020-08-14 | 桑格摩生物治疗股份有限公司 | 用于核酸酶介导的转基因靶向整合的方法和组合物 |
KR102247979B1 (ko) | 2012-05-25 | 2021-05-04 | 셀렉티스 | 면역요법을 위한 동종이형 및 면역억제제 저항성인 t 세포의 조작 방법 |
AU2013308770B2 (en) * | 2012-08-29 | 2019-01-17 | Sangamo Therapeutics, Inc. | Methods and compositions for treatment of a genetic condition |
AU2013355327A1 (en) | 2012-12-05 | 2015-06-11 | Sangamo Therapeutics, Inc. | Methods and compositions for regulation of metabolic disorders |
JP6392245B2 (ja) * | 2012-12-18 | 2018-09-19 | ノバルティス アーゲー | 遺伝子改変哺乳動物細胞における治療用タンパク質の産生 |
CN110540991B (zh) | 2013-03-15 | 2023-10-24 | 通用医疗公司 | 使用截短的引导RNA(tru-gRNA)提高RNA引导的基因组编辑的特异性 |
CN116083487A (zh) * | 2013-05-15 | 2023-05-09 | 桑格摩生物治疗股份有限公司 | 用于治疗遗传病状的方法和组合物 |
CA2920899C (en) | 2013-08-28 | 2023-02-28 | Sangamo Biosciences, Inc. | Compositions for linking dna-binding domains and cleavage domains |
DK3068881T3 (en) | 2013-11-13 | 2019-04-15 | Childrens Medical Center | NUCLEASE-MEDIATED REGULATION OF GENEPRESSION |
US9771403B2 (en) * | 2013-12-09 | 2017-09-26 | Sangamo Therapeutics, Inc. | Methods and compositions for treating hemophilia |
US20170037431A1 (en) * | 2014-05-01 | 2017-02-09 | University Of Washington | In vivo Gene Engineering with Adenoviral Vectors |
US11110154B2 (en) | 2014-05-08 | 2021-09-07 | Sangamo Therapeutics, Inc. | Methods and compositions for treating Huntington's Disease |
US9816074B2 (en) * | 2014-07-25 | 2017-11-14 | Sangamo Therapeutics, Inc. | Methods and compositions for modulating nuclease-mediated genome engineering in hematopoietic stem cells |
EP3054007A1 (en) | 2015-02-09 | 2016-08-10 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Recombinant adeno-associated virus particle purification comprising an immuno-affinity purification step |
IL284707B2 (en) * | 2015-05-12 | 2024-01-01 | Sangamo Therapeutics Inc | Regulation of nuclease-mediated gene expression |
US9957501B2 (en) * | 2015-06-18 | 2018-05-01 | Sangamo Therapeutics, Inc. | Nuclease-mediated regulation of gene expression |
EP3322297A4 (en) | 2015-07-13 | 2019-04-17 | Sangamo Therapeutics, Inc. | RELEASE METHOD AND COMPOSITIONS FOR NUCLEASE-DERIVED GENOMINE ENGINEERING |
EP3504327B1 (en) | 2016-08-24 | 2021-10-06 | Sangamo Therapeutics, Inc. | Engineered target specific nucleases |
PL3504229T3 (pl) | 2016-08-24 | 2022-04-19 | Sangamo Therapeutics, Inc. | Regulacja ekspresji genów przy użyciu nukleaz poddanych inżynierii |
US20180185516A1 (en) | 2016-12-09 | 2018-07-05 | Sangamo Therapeutics, Inc. | Delivery of target specific nucleases |
EP3665271A4 (en) | 2017-08-08 | 2021-04-28 | Sangamo Therapeutics, Inc. | CELL TARGETING MEDIA BY A CHEMICAL ANTIGEN RECEPTOR |
-
2017
- 2017-08-24 PL PL17844404T patent/PL3504229T3/pl unknown
- 2017-08-24 SG SG10201913315SA patent/SG10201913315SA/en unknown
- 2017-08-24 WO PCT/US2017/048397 patent/WO2018039440A1/en active Application Filing
- 2017-08-24 JP JP2019510787A patent/JP7203014B2/ja active Active
- 2017-08-24 AU AU2017315406A patent/AU2017315406B2/en active Active
- 2017-08-24 CN CN201780050637.3A patent/CN110325635B/zh active Active
- 2017-08-24 NZ NZ750938A patent/NZ750938A/en unknown
- 2017-08-24 DK DK17844404.8T patent/DK3504229T3/da active
- 2017-08-24 EP EP17844404.8A patent/EP3504229B1/en active Active
- 2017-08-24 MX MX2019002207A patent/MX2019002207A/es unknown
- 2017-08-24 SG SG11201901531TA patent/SG11201901531TA/en unknown
- 2017-08-24 HU HUE17844404A patent/HUE056707T2/hu unknown
- 2017-08-24 HR HRP20212025TT patent/HRP20212025T1/hr unknown
- 2017-08-24 RS RS20211591A patent/RS62758B1/sr unknown
- 2017-08-24 ES ES17844404T patent/ES2900317T3/es active Active
- 2017-08-24 TN TNP/2019/000048A patent/TN2019000048A1/en unknown
- 2017-08-24 LT LTEPPCT/US2017/048397T patent/LT3504229T/lt unknown
- 2017-08-24 IL IL264639A patent/IL264639B2/en unknown
- 2017-08-24 KR KR1020197005015A patent/KR102572759B1/ko active IP Right Grant
- 2017-08-24 BR BR112019003100A patent/BR112019003100A2/pt unknown
- 2017-08-24 MY MYPI2019000673A patent/MY193234A/en unknown
- 2017-08-24 EP EP21198842.3A patent/EP3995574A1/en not_active Withdrawn
- 2017-08-24 PT PT178444048T patent/PT3504229T/pt unknown
- 2017-08-24 SI SI201731029T patent/SI3504229T1/sl unknown
- 2017-08-24 PE PE2019000410A patent/PE20190568A1/es unknown
- 2017-08-24 CA CA3034882A patent/CA3034882A1/en active Pending
- 2017-08-24 CR CR20190098A patent/CR20190098A/es unknown
-
2019
- 2019-02-06 ZA ZA2019/00765A patent/ZA201900765B/en unknown
- 2019-02-08 PH PH12019500281A patent/PH12019500281A1/en unknown
- 2019-02-14 DO DO2019000031A patent/DOP2019000031A/es unknown
- 2019-02-14 CL CL2019000399A patent/CL2019000399A1/es unknown
- 2019-02-15 CO CONC2019/0001364A patent/CO2019001364A2/es unknown
- 2019-02-21 US US16/281,751 patent/US10563184B2/en active Active
- 2019-03-12 EC ECSENADI201917082A patent/ECSP19017082A/es unknown
-
2020
- 2020-01-09 US US16/738,766 patent/US11845965B2/en active Active
-
2021
- 2021-06-29 AU AU2021204432A patent/AU2021204432B2/en active Active
Also Published As
Similar Documents
Publication | Publication Date | Title |
---|---|---|
HRP20212025T1 (hr) | Regulacija ekspresije gena pomoću projektiranih nukleaza | |
Schöler | The potential of stem cells: An inventory | |
JP2019524150A5 (hr) | ||
CN105039339B (zh) | 一种以RNA介导的特异性敲除绵羊FecB基因的方法及其专用sgRNA | |
CN109715198A (zh) | 用于治疗血红蛋白病的材料和方法 | |
JP2017532955A5 (hr) | ||
MX2018008733A (es) | Metodos para extender la capacidad replicativa de las celulas somaticas durante un proceso de cultivo ex vivo. | |
CN109312308A (zh) | 通过使用核酸酶来进行人神经干细胞的基因组编辑 | |
JP2019500043A5 (hr) | ||
CN108513546A (zh) | 用于治疗杜氏肌营养不良症的材料和方法 | |
JP2019508051A5 (hr) | ||
JP2017035113A5 (hr) | ||
Stocum et al. | Stretching the limits: stem cells in regeneration science | |
IL308706A (en) | Preparations and methods for the treatment of hemoglobinopathies | |
RU2019127919A (ru) | Композиции и способы, предназначенные для лечения гемоглобинопатий | |
JP2019520844A5 (hr) | ||
CN107287245A (zh) | 一种基于CRISPR/Cas9技术的Glrx1基因敲除动物模型的构建方法 | |
CN108441520A (zh) | 利用CRISPR/Cas9系统构建的基因条件性敲除方法 | |
JP2017513477A5 (hr) | ||
CN109328231A (zh) | 用于治疗1a型糖原贮积病的材料和方法 | |
WO2012167192A3 (en) | Methods and products for producing engineered mammalian cell lines with amplified transgenes | |
CN104531705A (zh) | 利用CRISPR-Cas9系统敲除动物myostatin基因的方法 | |
Grillo et al. | Old questions, new models: unraveling complex organ regeneration with new experimental approaches | |
WO2021083183A9 (zh) | 一种造血干细胞hbb基因修复的方法及产品 | |
CN111629747A (zh) | Crispr-cas9修饰的cd34+人血色素干细胞和祖细胞及其用途 |