AU2016287836A1 - CRISPR/Cas9-based treatments - Google Patents
CRISPR/Cas9-based treatments Download PDFInfo
- Publication number
- AU2016287836A1 AU2016287836A1 AU2016287836A AU2016287836A AU2016287836A1 AU 2016287836 A1 AU2016287836 A1 AU 2016287836A1 AU 2016287836 A AU2016287836 A AU 2016287836A AU 2016287836 A AU2016287836 A AU 2016287836A AU 2016287836 A1 AU2016287836 A1 AU 2016287836A1
- Authority
- AU
- Australia
- Prior art keywords
- seq
- dystrophy
- corneal dystrophy
- corneal
- pct
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Abandoned
Links
- 108091033409 CRISPR Proteins 0.000 title claims description 31
- 238000011282 treatment Methods 0.000 title description 5
- 238000010354 CRISPR gene editing Methods 0.000 title 1
- 208000003923 Hereditary Corneal Dystrophies Diseases 0.000 claims abstract description 48
- 238000000034 method Methods 0.000 claims abstract description 45
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims abstract description 44
- 101710163270 Nuclease Proteins 0.000 claims abstract description 43
- 108020004414 DNA Proteins 0.000 claims abstract description 39
- 108091092878 Microsatellite Proteins 0.000 claims abstract description 38
- 201000010099 disease Diseases 0.000 claims abstract description 29
- 230000008439 repair process Effects 0.000 claims abstract description 22
- 230000006780 non-homologous end joining Effects 0.000 claims abstract description 20
- 230000034431 double-strand break repair via homologous recombination Effects 0.000 claims abstract description 13
- 238000003776 cleavage reaction Methods 0.000 claims abstract description 8
- 230000007017 scission Effects 0.000 claims abstract description 8
- 102100023967 Keratin, type I cytoskeletal 12 Human genes 0.000 claims description 78
- 206010011005 corneal dystrophy Diseases 0.000 claims description 53
- 208000009415 Spinocerebellar Ataxias Diseases 0.000 claims description 32
- 108090000623 proteins and genes Proteins 0.000 claims description 19
- 150000007523 nucleic acids Chemical class 0.000 claims description 18
- 201000004224 Schnyder corneal dystrophy Diseases 0.000 claims description 15
- 208000035475 disorder Diseases 0.000 claims description 14
- 201000003775 lattice corneal dystrophy Diseases 0.000 claims description 14
- 102000039446 nucleic acids Human genes 0.000 claims description 14
- 108020004707 nucleic acids Proteins 0.000 claims description 14
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 13
- 206010064571 Gene mutation Diseases 0.000 claims description 13
- 239000013604 expression vector Substances 0.000 claims description 13
- 210000001519 tissue Anatomy 0.000 claims description 13
- 208000036626 Mental retardation Diseases 0.000 claims description 12
- 208000033051 Fuchs endothelial corneal dystrophy Diseases 0.000 claims description 11
- 210000004087 cornea Anatomy 0.000 claims description 11
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 10
- 102000007370 Ataxin2 Human genes 0.000 claims description 9
- 108010032951 Ataxin2 Proteins 0.000 claims description 9
- 208000002569 Machado-Joseph Disease Diseases 0.000 claims description 9
- 208000036834 Spinocerebellar ataxia type 3 Diseases 0.000 claims description 9
- 201000003629 Spinocerebellar ataxia type 8 Diseases 0.000 claims description 9
- 201000003594 spinocerebellar ataxia type 12 Diseases 0.000 claims description 9
- 102100032187 Androgen receptor Human genes 0.000 claims description 8
- 208000024412 Friedreich ataxia Diseases 0.000 claims description 8
- 101000775732 Homo sapiens Androgen receptor Proteins 0.000 claims description 8
- 208000023105 Huntington disease Diseases 0.000 claims description 8
- 201000003622 Spinocerebellar ataxia type 2 Diseases 0.000 claims description 8
- 201000003620 Spinocerebellar ataxia type 6 Diseases 0.000 claims description 8
- 208000006269 X-Linked Bulbo-Spinal Atrophy Diseases 0.000 claims description 8
- 201000004889 corneal granular dystrophy Diseases 0.000 claims description 8
- 201000003415 fragile X-associated tremor/ataxia syndrome Diseases 0.000 claims description 8
- 208000014706 granular corneal dystrophy Diseases 0.000 claims description 8
- 238000002347 injection Methods 0.000 claims description 8
- 239000007924 injection Substances 0.000 claims description 8
- 201000003624 spinocerebellar ataxia type 1 Diseases 0.000 claims description 8
- 201000003570 spinocerebellar ataxia type 17 Diseases 0.000 claims description 8
- 201000003632 spinocerebellar ataxia type 7 Diseases 0.000 claims description 8
- 201000004182 Congenital stromal corneal dystrophy Diseases 0.000 claims description 7
- 230000003511 endothelial effect Effects 0.000 claims description 7
- 102000004169 proteins and genes Human genes 0.000 claims description 7
- 102100024378 AF4/FMR2 family member 2 Human genes 0.000 claims description 6
- 102000007371 Ataxin-3 Human genes 0.000 claims description 6
- 101000833172 Homo sapiens AF4/FMR2 family member 2 Proteins 0.000 claims description 6
- 101000828537 Homo sapiens Synaptic functional regulator FMR1 Proteins 0.000 claims description 6
- 102100023532 Synaptic functional regulator FMR1 Human genes 0.000 claims description 6
- 239000000203 mixture Substances 0.000 claims description 6
- 102000053602 DNA Human genes 0.000 claims description 5
- 201000004173 Epithelial basement membrane dystrophy Diseases 0.000 claims description 5
- 201000004225 Thiel-Behnke corneal dystrophy Diseases 0.000 claims description 5
- 208000009624 holoprosencephaly Diseases 0.000 claims description 5
- 206010005155 Blepharophimosis Diseases 0.000 claims description 4
- 201000000304 Cleidocranial dysplasia Diseases 0.000 claims description 4
- 206010066131 Congenital central hypoventilation syndrome Diseases 0.000 claims description 4
- 206010015995 Eyelid ptosis Diseases 0.000 claims description 4
- 208000005417 Fleck corneal dystrophy Diseases 0.000 claims description 4
- 208000001914 Fragile X syndrome Diseases 0.000 claims description 4
- 206010056438 Growth hormone deficiency Diseases 0.000 claims description 4
- 208000016031 Haddad syndrome Diseases 0.000 claims description 4
- 206010072361 Hand-foot-genital syndrome Diseases 0.000 claims description 4
- 206010021750 Infantile Spasms Diseases 0.000 claims description 4
- 208000035899 Infantile spasms syndrome Diseases 0.000 claims description 4
- 206010068871 Myotonic dystrophy Diseases 0.000 claims description 4
- 201000009110 Oculopharyngeal muscular dystrophy Diseases 0.000 claims description 4
- 201000003692 Partington syndrome Diseases 0.000 claims description 4
- 201000004223 Reis-Bucklers corneal dystrophy Diseases 0.000 claims description 4
- 206010042778 Syndactyly Diseases 0.000 claims description 4
- 201000006791 West syndrome Diseases 0.000 claims description 4
- 206010015037 epilepsy Diseases 0.000 claims description 4
- 208000001901 epithelial recurrent erosion dystrophy Diseases 0.000 claims description 4
- 210000000744 eyelid Anatomy 0.000 claims description 4
- 230000008826 genomic mutation Effects 0.000 claims description 4
- 201000004207 posterior polymorphous corneal dystrophy Diseases 0.000 claims description 4
- 201000003004 ptosis Diseases 0.000 claims description 4
- 210000003786 sclera Anatomy 0.000 claims description 4
- 201000001925 Fuchs' endothelial dystrophy Diseases 0.000 claims description 3
- 208000006335 Lisch epithelial corneal dystrophy Diseases 0.000 claims description 3
- 201000004219 Meesmann corneal dystrophy Diseases 0.000 claims description 3
- 201000004216 Posterior amorphous corneal dystrophy Diseases 0.000 claims description 3
- 206010002022 amyloidosis Diseases 0.000 claims description 3
- 238000009472 formulation Methods 0.000 claims description 3
- 208000021907 Central cloudy dystrophy of François Diseases 0.000 claims description 2
- 206010010356 Congenital anomaly Diseases 0.000 claims description 2
- 201000004176 Gelatinous drop-like corneal dystrophy Diseases 0.000 claims description 2
- 101000854936 Homo sapiens Visual system homeobox 1 Proteins 0.000 claims description 2
- 208000002615 Juvenile Epithelial of Meesmann Corneal Dystrophy Diseases 0.000 claims description 2
- 102100020673 Visual system homeobox 1 Human genes 0.000 claims description 2
- 238000002513 implantation Methods 0.000 claims description 2
- 201000005139 macular corneal dystrophy Diseases 0.000 claims description 2
- 208000012584 pre-descemet corneal dystrophy Diseases 0.000 claims description 2
- 208000009996 subepithelial mucinous corneal dystrophy Diseases 0.000 claims description 2
- 238000010356 CRISPR-Cas9 genome editing Methods 0.000 claims 4
- 201000004175 X-linked endothelial corneal dystrophy Diseases 0.000 claims 1
- 101000975472 Homo sapiens Keratin, type I cytoskeletal 12 Proteins 0.000 description 77
- 101000894525 Homo sapiens Transforming growth factor-beta-induced protein ig-h3 Proteins 0.000 description 71
- 102100021398 Transforming growth factor-beta-induced protein ig-h3 Human genes 0.000 description 71
- 108020005004 Guide RNA Proteins 0.000 description 49
- 102100039547 UbiA prenyltransferase domain-containing protein 1 Human genes 0.000 description 30
- 101001056469 Homo sapiens Keratin, type II cytoskeletal 3 Proteins 0.000 description 20
- 101000608653 Homo sapiens UbiA prenyltransferase domain-containing protein 1 Proteins 0.000 description 20
- 102100025759 Keratin, type II cytoskeletal 3 Human genes 0.000 description 20
- 102100038028 1-phosphatidylinositol 3-phosphate 5-kinase Human genes 0.000 description 18
- 101100174573 Homo sapiens PIKFYVE gene Proteins 0.000 description 18
- 230000035772 mutation Effects 0.000 description 18
- 102100035784 Decorin Human genes 0.000 description 16
- 102100040496 Collagen alpha-2(VIII) chain Human genes 0.000 description 12
- 101000749886 Homo sapiens Collagen alpha-2(VIII) chain Proteins 0.000 description 12
- 210000004027 cell Anatomy 0.000 description 12
- 108010048992 Transcription Factor 4 Proteins 0.000 description 9
- 102100023489 Transcription factor 4 Human genes 0.000 description 9
- 230000008685 targeting Effects 0.000 description 8
- 101150017815 TCF4 gene Proteins 0.000 description 6
- 241000282485 Vulpes vulpes Species 0.000 description 6
- 238000013459 approach Methods 0.000 description 6
- 102200088796 rs118020901 Human genes 0.000 description 6
- 102200059672 rs118203949 Human genes 0.000 description 6
- 102200059673 rs118203953 Human genes 0.000 description 6
- 102220000690 rs121918337 Human genes 0.000 description 6
- 102220024006 rs397515545 Human genes 0.000 description 6
- 102200089582 rs5030804 Human genes 0.000 description 6
- 102200004603 rs58343600 Human genes 0.000 description 6
- 102220005858 rs80338741 Human genes 0.000 description 6
- 102220006442 rs80338742 Human genes 0.000 description 6
- 210000003462 vein Anatomy 0.000 description 6
- 208000035250 cutaneous malignant susceptibility to 1 melanoma Diseases 0.000 description 5
- 238000012239 gene modification Methods 0.000 description 5
- 238000010362 genome editing Methods 0.000 description 5
- 201000001441 melanoma Diseases 0.000 description 5
- 102200040258 rs121909208 Human genes 0.000 description 5
- 102200040253 rs121909209 Human genes 0.000 description 5
- 238000001415 gene therapy Methods 0.000 description 4
- 238000011002 quantification Methods 0.000 description 4
- 102220257523 rs1114167731 Human genes 0.000 description 4
- 102200040436 rs121909211 Human genes 0.000 description 4
- 102200040244 rs121909215 Human genes 0.000 description 4
- 102220024596 rs267607431 Human genes 0.000 description 4
- 102200004576 rs58162394 Human genes 0.000 description 4
- 230000009885 systemic effect Effects 0.000 description 4
- 238000001890 transfection Methods 0.000 description 4
- 238000011144 upstream manufacturing Methods 0.000 description 4
- 108700028369 Alleles Proteins 0.000 description 3
- 108091093088 Amplicon Proteins 0.000 description 3
- 101000785626 Homo sapiens Zinc finger E-box-binding homeobox 1 Proteins 0.000 description 3
- 241000193996 Streptococcus pyogenes Species 0.000 description 3
- 102100026457 Zinc finger E-box-binding homeobox 1 Human genes 0.000 description 3
- 210000001367 artery Anatomy 0.000 description 3
- 208000028485 lattice corneal dystrophy type I Diseases 0.000 description 3
- 238000012986 modification Methods 0.000 description 3
- 230000004048 modification Effects 0.000 description 3
- 239000013612 plasmid Substances 0.000 description 3
- 102200004565 rs11650915 Human genes 0.000 description 3
- 102200059610 rs118203945 Human genes 0.000 description 3
- 102200059669 rs118203946 Human genes 0.000 description 3
- 102200059615 rs118203947 Human genes 0.000 description 3
- 102200059663 rs118203948 Human genes 0.000 description 3
- 102200059611 rs118203950 Human genes 0.000 description 3
- 102200059492 rs118203951 Human genes 0.000 description 3
- 102200059666 rs118203952 Human genes 0.000 description 3
- 102200040434 rs121909210 Human genes 0.000 description 3
- 102200040404 rs121909210 Human genes 0.000 description 3
- 102200040401 rs121909212 Human genes 0.000 description 3
- 102200040397 rs121909214 Human genes 0.000 description 3
- 102200040403 rs121909216 Human genes 0.000 description 3
- 102200040225 rs121909217 Human genes 0.000 description 3
- 102200012808 rs121918336 Human genes 0.000 description 3
- 102200161233 rs137853169 Human genes 0.000 description 3
- 102220146320 rs141986556 Human genes 0.000 description 3
- 102220024226 rs200258980 Human genes 0.000 description 3
- 102200040393 rs267607110 Human genes 0.000 description 3
- 102200004717 rs267607386 Human genes 0.000 description 3
- 102200004568 rs267607387 Human genes 0.000 description 3
- 102200004567 rs28936695 Human genes 0.000 description 3
- 102200059668 rs397514669 Human genes 0.000 description 3
- 102220023646 rs397515067 Human genes 0.000 description 3
- 102200004548 rs57218384 Human genes 0.000 description 3
- 102200053908 rs57872071 Human genes 0.000 description 3
- 102200004598 rs58038639 Human genes 0.000 description 3
- 102200004553 rs58410481 Human genes 0.000 description 3
- 102220035853 rs587777258 Human genes 0.000 description 3
- 102200004569 rs58864803 Human genes 0.000 description 3
- 102200004601 rs58918655 Human genes 0.000 description 3
- 102200004582 rs59202432 Human genes 0.000 description 3
- 102200004607 rs59350319 Human genes 0.000 description 3
- 102220024224 rs59465138 Human genes 0.000 description 3
- 102200053910 rs60410063 Human genes 0.000 description 3
- 102220024225 rs61167390 Human genes 0.000 description 3
- 102200004596 rs61282718 Human genes 0.000 description 3
- 102220024223 rs62065925 Human genes 0.000 description 3
- 102200004578 rs62635290 Human genes 0.000 description 3
- 102200028488 rs782308462 Human genes 0.000 description 3
- 102200030577 rs80358191 Human genes 0.000 description 3
- 102220005941 rs80358192 Human genes 0.000 description 3
- 102220264073 rs951997505 Human genes 0.000 description 3
- 238000010453 CRISPR/Cas method Methods 0.000 description 2
- 201000008163 Dentatorubral pallidoluysian atrophy Diseases 0.000 description 2
- 108010008532 Deoxyribonuclease I Proteins 0.000 description 2
- 102000007260 Deoxyribonuclease I Human genes 0.000 description 2
- 206010016202 Familial Amyloidosis Diseases 0.000 description 2
- 208000036861 Fuchs endothelial 1 corneal dystrophy Diseases 0.000 description 2
- 101100174574 Mus musculus Pikfyve gene Proteins 0.000 description 2
- 102000009134 Steryl-Sulfatase Human genes 0.000 description 2
- 108010087999 Steryl-Sulfatase Proteins 0.000 description 2
- 208000021332 Syndactyly type 2 Diseases 0.000 description 2
- 208000001001 X-linked ichthyosis Diseases 0.000 description 2
- 101100323865 Xenopus laevis arg1 gene Proteins 0.000 description 2
- 101150063416 add gene Proteins 0.000 description 2
- 230000002146 bilateral effect Effects 0.000 description 2
- 239000003795 chemical substances by application Substances 0.000 description 2
- 238000012937 correction Methods 0.000 description 2
- 230000007812 deficiency Effects 0.000 description 2
- 238000012217 deletion Methods 0.000 description 2
- 230000037430 deletion Effects 0.000 description 2
- 230000007613 environmental effect Effects 0.000 description 2
- 208000030533 eye disease Diseases 0.000 description 2
- 230000009395 genetic defect Effects 0.000 description 2
- 208000035474 group of disease Diseases 0.000 description 2
- 206010021198 ichthyosis Diseases 0.000 description 2
- 239000007943 implant Substances 0.000 description 2
- 230000002779 inactivation Effects 0.000 description 2
- 238000007912 intraperitoneal administration Methods 0.000 description 2
- 230000002601 intratumoral effect Effects 0.000 description 2
- 238000001990 intravenous administration Methods 0.000 description 2
- 210000004379 membrane Anatomy 0.000 description 2
- 239000012528 membrane Substances 0.000 description 2
- 210000001758 mesenteric vein Anatomy 0.000 description 2
- 239000002773 nucleotide Substances 0.000 description 2
- 125000003729 nucleotide group Chemical group 0.000 description 2
- 210000000056 organ Anatomy 0.000 description 2
- 230000002093 peripheral effect Effects 0.000 description 2
- 230000000750 progressive effect Effects 0.000 description 2
- 208000026079 recessive X-linked ichthyosis Diseases 0.000 description 2
- 102200040405 rs121909211 Human genes 0.000 description 2
- 102220011837 rs387907335 Human genes 0.000 description 2
- 201000002957 synpolydactyly Diseases 0.000 description 2
- 102000007372 Ataxin-1 Human genes 0.000 description 1
- 108010032963 Ataxin-1 Proteins 0.000 description 1
- 108010032947 Ataxin-3 Proteins 0.000 description 1
- 102000007368 Ataxin-7 Human genes 0.000 description 1
- 108010032953 Ataxin-7 Proteins 0.000 description 1
- 102100020741 Atrophin-1 Human genes 0.000 description 1
- 201000004569 Blindness Diseases 0.000 description 1
- 208000034717 Congenital hereditary endothelial dystrophy type II Diseases 0.000 description 1
- 102000015775 Core Binding Factor Alpha 1 Subunit Human genes 0.000 description 1
- 108010024682 Core Binding Factor Alpha 1 Subunit Proteins 0.000 description 1
- 108700024394 Exon Proteins 0.000 description 1
- 201000007888 Finnish type amyloidosis Diseases 0.000 description 1
- 108010010285 Forkhead Box Protein L2 Proteins 0.000 description 1
- 102100035137 Forkhead box protein L2 Human genes 0.000 description 1
- 102000004878 Gelsolin Human genes 0.000 description 1
- 108090001064 Gelsolin Proteins 0.000 description 1
- 208000031518 Genetic corneal dystrophy Diseases 0.000 description 1
- 101000785083 Homo sapiens Atrophin-1 Proteins 0.000 description 1
- 101000692768 Homo sapiens Paired mesoderm homeobox protein 2B Proteins 0.000 description 1
- 101000609211 Homo sapiens Polyadenylate-binding protein 2 Proteins 0.000 description 1
- 101000915806 Homo sapiens Serine/threonine-protein phosphatase 2A 55 kDa regulatory subunit B beta isoform Proteins 0.000 description 1
- 101000687911 Homo sapiens Transcription factor SOX-3 Proteins 0.000 description 1
- 101000976643 Homo sapiens Zinc finger protein ZIC 2 Proteins 0.000 description 1
- 108091092195 Intron Proteins 0.000 description 1
- 206010025421 Macule Diseases 0.000 description 1
- 108010052185 Myotonin-Protein Kinase Proteins 0.000 description 1
- 102100022437 Myotonin-protein kinase Human genes 0.000 description 1
- 206010028980 Neoplasm Diseases 0.000 description 1
- 241000083652 Osca Species 0.000 description 1
- 102100026354 Paired mesoderm homeobox protein 2B Human genes 0.000 description 1
- 102100039427 Polyadenylate-binding protein 2 Human genes 0.000 description 1
- 102100029014 Serine/threonine-protein phosphatase 2A 55 kDa regulatory subunit B beta isoform Human genes 0.000 description 1
- 102100024276 Transcription factor SOX-3 Human genes 0.000 description 1
- 102100023492 Zinc finger protein ZIC 2 Human genes 0.000 description 1
- 239000002253 acid Substances 0.000 description 1
- 239000000980 acid dye Substances 0.000 description 1
- 125000003275 alpha amino acid group Chemical group 0.000 description 1
- 230000003321 amplification Effects 0.000 description 1
- 210000002376 aorta thoracic Anatomy 0.000 description 1
- 210000002565 arteriole Anatomy 0.000 description 1
- 238000003556 assay Methods 0.000 description 1
- 230000015572 biosynthetic process Effects 0.000 description 1
- 210000002302 brachial artery Anatomy 0.000 description 1
- 210000004004 carotid artery internal Anatomy 0.000 description 1
- 230000007248 cellular mechanism Effects 0.000 description 1
- 238000006243 chemical reaction Methods 0.000 description 1
- 210000003287 ciliary artery Anatomy 0.000 description 1
- 230000000295 complement effect Effects 0.000 description 1
- 210000000795 conjunctiva Anatomy 0.000 description 1
- 210000004351 coronary vessel Anatomy 0.000 description 1
- 210000000695 crystalline len Anatomy 0.000 description 1
- 238000011257 definitive treatment Methods 0.000 description 1
- 229910003460 diamond Inorganic materials 0.000 description 1
- 239000010432 diamond Substances 0.000 description 1
- 239000003814 drug Substances 0.000 description 1
- 229940079593 drug Drugs 0.000 description 1
- 210000003527 eukaryotic cell Anatomy 0.000 description 1
- 210000001105 femoral artery Anatomy 0.000 description 1
- 210000003191 femoral vein Anatomy 0.000 description 1
- 239000000835 fiber Substances 0.000 description 1
- 239000012634 fragment Substances 0.000 description 1
- 230000002496 gastric effect Effects 0.000 description 1
- 239000010931 gold Substances 0.000 description 1
- 229910052737 gold Inorganic materials 0.000 description 1
- 208000028431 granular corneal dystrophy 2 Diseases 0.000 description 1
- 201000004183 granular corneal dystrophy type II Diseases 0.000 description 1
- 210000002989 hepatic vein Anatomy 0.000 description 1
- 210000005260 human cell Anatomy 0.000 description 1
- 238000003780 insertion Methods 0.000 description 1
- 230000037431 insertion Effects 0.000 description 1
- 238000001361 intraarterial administration Methods 0.000 description 1
- 238000007918 intramuscular administration Methods 0.000 description 1
- 239000007927 intramuscular injection Substances 0.000 description 1
- 238000010255 intramuscular injection Methods 0.000 description 1
- 238000007913 intrathecal administration Methods 0.000 description 1
- 210000000554 iris Anatomy 0.000 description 1
- 210000004731 jugular vein Anatomy 0.000 description 1
- 210000004962 mammalian cell Anatomy 0.000 description 1
- 238000003199 nucleic acid amplification method Methods 0.000 description 1
- 210000001328 optic nerve Anatomy 0.000 description 1
- 230000010412 perfusion Effects 0.000 description 1
- 210000003240 portal vein Anatomy 0.000 description 1
- 201000008526 posterior polymorphous corneal dystrophy 1 Diseases 0.000 description 1
- 210000001147 pulmonary artery Anatomy 0.000 description 1
- 210000003492 pulmonary vein Anatomy 0.000 description 1
- 210000001747 pupil Anatomy 0.000 description 1
- 230000009467 reduction Effects 0.000 description 1
- 238000009877 rendering Methods 0.000 description 1
- 210000001525 retina Anatomy 0.000 description 1
- 238000012552 review Methods 0.000 description 1
- 102200040392 rs267607109 Human genes 0.000 description 1
- 210000003752 saphenous vein Anatomy 0.000 description 1
- 230000003007 single stranded DNA break Effects 0.000 description 1
- 210000000955 splenic vein Anatomy 0.000 description 1
- 238000007920 subcutaneous administration Methods 0.000 description 1
- 239000007929 subcutaneous injection Substances 0.000 description 1
- 238000010254 subcutaneous injection Methods 0.000 description 1
- 238000006467 substitution reaction Methods 0.000 description 1
- 238000007910 systemic administration Methods 0.000 description 1
- 238000002560 therapeutic procedure Methods 0.000 description 1
- 238000002054 transplantation Methods 0.000 description 1
- 210000005166 vasculature Anatomy 0.000 description 1
- 210000001631 vena cava inferior Anatomy 0.000 description 1
- 210000002620 vena cava superior Anatomy 0.000 description 1
- 230000004393 visual impairment Effects 0.000 description 1
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
- A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/08—Antiepileptics; Anticonvulsants
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
-
- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
Landscapes
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Mycology (AREA)
- Ophthalmology & Optometry (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Epidemiology (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Rheumatology (AREA)
- Pulmonology (AREA)
- Pain & Pain Management (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562188013P | 2015-07-02 | 2015-07-02 | |
| US62/188,013 | 2015-07-02 | ||
| PCT/US2016/040962 WO2017004616A1 (en) | 2015-07-02 | 2016-07-05 | Crispr/cas9-based treatments |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AU2016287836A1 true AU2016287836A1 (en) | 2018-02-15 |
Family
ID=57609222
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2016287836A Abandoned AU2016287836A1 (en) | 2015-07-02 | 2016-07-05 | CRISPR/Cas9-based treatments |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US20200010854A1 (https=) |
| EP (1) | EP3317409A4 (https=) |
| JP (1) | JP2018520149A (https=) |
| KR (1) | KR20180041120A (https=) |
| CN (1) | CN108350446A (https=) |
| AU (1) | AU2016287836A1 (https=) |
| BR (1) | BR112017028201A2 (https=) |
| CA (1) | CA2989331A1 (https=) |
| CL (1) | CL2017003411A1 (https=) |
| EA (1) | EA201890203A1 (https=) |
| IL (1) | IL256279A (https=) |
| MX (1) | MX2017016921A (https=) |
| WO (1) | WO2017004616A1 (https=) |
Families Citing this family (48)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10323236B2 (en) | 2011-07-22 | 2019-06-18 | 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 |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| ES2788426T3 (es) | 2014-06-16 | 2020-10-21 | Univ Johns Hopkins | Composiciones y métodos para la expresión de ARNs de guía de CRISPR utilizando el promotor de H1 |
| US10077453B2 (en) | 2014-07-30 | 2018-09-18 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
| IL310721B2 (en) | 2015-10-23 | 2025-11-01 | Harvard College | Nucleobase editors and uses thereof |
| EP4036228A1 (en) | 2015-11-13 | 2022-08-03 | Avellino Lab USA, Inc. | Methods for the treatment of corneal dystrophies |
| KR20180134412A (ko) * | 2016-04-22 | 2018-12-18 | 인텔리아 테라퓨틱스, 인크. | 전사 인자 4에서 트라이뉴클레오타이드 반복부와 연관된 질환의 치료를 위한 조성물 및 방법 |
| CN110214183A (zh) | 2016-08-03 | 2019-09-06 | 哈佛大学的校长及成员们 | 腺苷核碱基编辑器及其用途 |
| WO2018031683A1 (en) | 2016-08-09 | 2018-02-15 | President And Fellows Of Harvard College | Programmable cas9-recombinase fusion proteins and uses thereof |
| WO2020046861A1 (en) * | 2018-08-27 | 2020-03-05 | Avellino Lab Usa, Inc. | Crispr/cas9 systems, and methods of use thereof |
| US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| KR102622411B1 (ko) | 2016-10-14 | 2024-01-10 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵염기 에디터의 aav 전달 |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| US12390514B2 (en) | 2017-03-09 | 2025-08-19 | President And Fellows Of Harvard College | Cancer vaccine |
| EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
| US11542496B2 (en) | 2017-03-10 | 2023-01-03 | President And Fellows Of Harvard College | Cytosine to guanine base editor |
| WO2018165541A1 (en) | 2017-03-10 | 2018-09-13 | The Board Of Regents Of The University Of Texas System | Treatment of fuchs' endothelial corneal dystrophy |
| KR20240116572A (ko) | 2017-03-23 | 2024-07-29 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제 |
| US11560566B2 (en) | 2017-05-12 | 2023-01-24 | President And Fellows Of Harvard College | Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation |
| WO2018225807A1 (ja) * | 2017-06-07 | 2018-12-13 | 国立大学法人東京大学 | 顆粒状角膜変性症に対する遺伝子治療薬 |
| CN111801345A (zh) | 2017-07-28 | 2020-10-20 | 哈佛大学的校长及成员们 | 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物 |
| EP3676376B1 (en) | 2017-08-30 | 2025-01-15 | President and Fellows of Harvard College | High efficiency base editors comprising gam |
| KR20250107288A (ko) | 2017-10-16 | 2025-07-11 | 더 브로드 인스티튜트, 인코퍼레이티드 | 아데노신 염기 편집제의 용도 |
| US12406749B2 (en) | 2017-12-15 | 2025-09-02 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| DE102017131324A1 (de) | 2017-12-27 | 2019-06-27 | Beckhoff Automation Gmbh | Statormodul und Planarantriebssystem |
| CN108949823B (zh) * | 2017-12-29 | 2020-02-14 | 广州医科大学附属第三医院（广州重症孕产妇救治中心、广州柔济医院） | 靶向敲除ATXN3基因中扩展突变型polyQ序列的方法 |
| EP3790980A4 (en) * | 2018-05-06 | 2022-03-23 | Emendobio Inc. | DIFFERENTIAL KNOCKOUT OF AN ALLEL OF A HETEROZYGOUS ELAN GENE |
| US12157760B2 (en) | 2018-05-23 | 2024-12-03 | The Broad Institute, Inc. | Base editors and uses thereof |
| US12522807B2 (en) | 2018-07-09 | 2026-01-13 | The Broad Institute, Inc. | RNA programmable epigenetic RNA modifiers and uses thereof |
| KR102126573B1 (ko) | 2018-10-18 | 2020-06-26 | 대한민국 | CRISPR-Cas9 시스템을 이용한 유색고약버섯의 리그닌 분해효소의 유전자 편집방법 및 이의 용도 |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| KR102929494B1 (ko) * | 2018-10-29 | 2026-02-20 | 차이나 어그리컬처럴 유니버시티 | 신규 CRISPR/Cas12f 효소 및 시스템 |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| WO2020191233A1 (en) | 2019-03-19 | 2020-09-24 | The Broad Institute, Inc. | Methods and compositions for editing nucleotide sequences |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| WO2021076883A2 (en) * | 2019-10-16 | 2021-04-22 | Brown University | Muscle regeneration and growth |
| IL297761A (en) | 2020-05-08 | 2022-12-01 | Broad Inst Inc | Methods and compositions for simultaneously editing two helices of a designated double-helix nucleotide sequence |
| CN111849991B (zh) * | 2020-08-05 | 2022-04-08 | 武汉纽福斯生物科技有限公司 | 一种寡核苷酸及其应用 |
| EP4430185A1 (en) * | 2021-11-09 | 2024-09-18 | Prime Medicine, Inc. | Genome editing compositions and methods for treatment of fuchs endothelial corneal dystrophy |
| WO2023092132A1 (en) * | 2021-11-22 | 2023-05-25 | Mammoth Biosciences, Inc. | Effector proteins and uses thereof |
| IL320664A (en) * | 2022-11-02 | 2025-07-01 | Emendobio Inc | Compositions and methods for transcription factor 4 (tcf4) repeat expansion excision |
| CN116286823A (zh) * | 2023-04-04 | 2023-06-23 | 尧唐(上海)生物科技有限公司 | 靶向TGFBI基因的sgRNA、载体及其应用 |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2014093655A2 (en) * | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains |
| WO2015048577A2 (en) * | 2013-09-27 | 2015-04-02 | Editas Medicine, Inc. | Crispr-related methods and compositions |
| JP6712948B2 (ja) * | 2013-12-12 | 2020-06-24 | ザ・ブロード・インスティテュート・インコーポレイテッド | 組成物、及びヌクレオチドリピート障害におけるｃｒｉｓｐｒ−ｃａｓ系の使用方法 |
| JP7103750B2 (ja) * | 2013-12-12 | 2022-07-20 | ザ・ブロード・インスティテュート・インコーポレイテッド | ゲノム編集のためのＣＲＩＳＰＲ－Ｃａｓ系及び組成物の送達、使用及び治療適用 |
| KR20180134412A (ko) * | 2016-04-22 | 2018-12-18 | 인텔리아 테라퓨틱스, 인크. | 전사 인자 4에서 트라이뉴클레오타이드 반복부와 연관된 질환의 치료를 위한 조성물 및 방법 |
-
2016
- 2016-07-05 CN CN201680050474.4A patent/CN108350446A/zh active Pending
- 2016-07-05 BR BR112017028201A patent/BR112017028201A2/pt not_active Application Discontinuation
- 2016-07-05 KR KR1020187002971A patent/KR20180041120A/ko not_active Withdrawn
- 2016-07-05 JP JP2017567629A patent/JP2018520149A/ja active Pending
- 2016-07-05 EP EP16818953.8A patent/EP3317409A4/en not_active Withdrawn
- 2016-07-05 US US15/741,444 patent/US20200010854A1/en not_active Abandoned
- 2016-07-05 AU AU2016287836A patent/AU2016287836A1/en not_active Abandoned
- 2016-07-05 EA EA201890203A patent/EA201890203A1/ru unknown
- 2016-07-05 MX MX2017016921A patent/MX2017016921A/es unknown
- 2016-07-05 CA CA2989331A patent/CA2989331A1/en not_active Abandoned
- 2016-07-05 WO PCT/US2016/040962 patent/WO2017004616A1/en not_active Ceased
-
2017
- 2017-12-12 IL IL256279A patent/IL256279A/en unknown
- 2017-12-27 CL CL2017003411A patent/CL2017003411A1/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| CA2989331A1 (en) | 2017-01-05 |
| US20200010854A1 (en) | 2020-01-09 |
| EA201890203A1 (ru) | 2018-07-31 |
| IL256279A (en) | 2018-02-28 |
| BR112017028201A2 (pt) | 2018-08-28 |
| CL2017003411A1 (es) | 2018-08-17 |
| WO2017004616A1 (en) | 2017-01-05 |
| EP3317409A1 (en) | 2018-05-09 |
| JP2018520149A (ja) | 2018-07-26 |
| MX2017016921A (es) | 2018-04-10 |
| KR20180041120A (ko) | 2018-04-23 |
| EP3317409A4 (en) | 2019-02-20 |
| CN108350446A (zh) | 2018-07-31 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| AU2016287836A1 (en) | CRISPR/Cas9-based treatments | |
| Tsai et al. | Clustered regularly interspaced short palindromic repeats-based genome surgery for the treatment of autosomal dominant retinitis pigmentosa | |
| US20230233651A1 (en) | Materials and methods for treatment of titin-based myopathies and other titinopathies | |
| US11497816B2 (en) | Compositions and methods for treating fragile X syndrome and related syndromes | |
| JP2019055990A (ja) | 疾患の処置のためのトリシクロ−ｄｎａアンチセンスオリゴヌクレオチド、組成物及び方法 | |
| JP2019500348A (ja) | 眼疾患の処置のための組成物および方法 | |
| CN114040974A (zh) | 用于高效rna反式剪接的三螺旋终止子 | |
| JP2025175283A (ja) | Ａｍｄの処置のためのｈｔｒａ１調節 | |
| WO2023163131A1 (ja) | フレームシフト変異に起因する疾患のための医薬 | |
| Hu et al. | In vivo adenine base editing ameliorates Rho-associated autosomal dominant retinitis pigmentosa | |
| JP2020528735A (ja) | 反復伸長変異のためのゲノム編集システム | |
| JP2022510673A (ja) | アンチセンスオリゴヌクレオチドは、ａｂｃａ４の異常スプライシングをレスキューする | |
| CN116334141A (zh) | 基于基因编辑的RHO-R135W-adRP基因编辑药物 | |
| WO2022072458A1 (en) | Crispr/cas9 targeted excision of the intronic ctg18.1 trinucleotide repeat expansion of tcf4 as a therapy in fuchs' endothelial corneal dystrophy | |
| EP3652310B1 (en) | Gene editing system for correcting splicing defects | |
| JP7848113B2 (ja) | 目的遺伝子の相同遺伝子を転写活性化する方法および生体外診断方法 | |
| Baruah et al. | Macular Corneal Dystrophy: Past | |
| KR20190037167A (ko) | 혈액응고인자 viii 유전자 역위 보정능의 유전자 가위 시스템으로 구성된 혈우병 치료용 조성물 | |
| Lam et al. | 680P Developing effective RNA editing approaches to target the nonsense mutation in the key functional region of DMD | |
| WO2023220732A1 (en) | Methods and systems for correcting mutations in prph2 | |
| AU2024355054A1 (en) | Crispr-cas9 based tools: potential novel therapeutic approaches for machado-joseph disease | |
| LLADO SANTAEULARIA | THERAPEUTIC GENOME EDITING IN RETINA AND LIVER | |
| EP4222254A1 (en) | Crispr/cas9 targeted excision of the intronic ctg18.1 trinucleotide repeat expansion of tcf4 as a therapy in fuchs' endothelial corneal dystrophy | |
| IT202000008014A1 (it) | RNA guida e loro usi | |
| HK40029591A (en) | Genome editing system for repeat expansion mutation |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| MK1 | Application lapsed section 142(2)(a) - no request for examination in relevant period |