AU2018320849A1 - Recombinant adeno-associated vectors - Google Patents
Recombinant adeno-associated vectors Download PDFInfo
- Publication number
- AU2018320849A1 AU2018320849A1 AU2018320849A AU2018320849A AU2018320849A1 AU 2018320849 A1 AU2018320849 A1 AU 2018320849A1 AU 2018320849 A AU2018320849 A AU 2018320849A AU 2018320849 A AU2018320849 A AU 2018320849A AU 2018320849 A1 AU2018320849 A1 AU 2018320849A1
- Authority
- AU
- Australia
- Prior art keywords
- vectors
- raavrec3
- aav
- nucleic acid
- cell
- 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
- 239000013598 vector Substances 0.000 title claims abstract description 88
- 108090000623 proteins and genes Proteins 0.000 claims abstract description 58
- 241000702421 Dependoparvovirus Species 0.000 claims abstract description 9
- 239000000546 pharmaceutical excipient Substances 0.000 claims abstract description 7
- 239000003085 diluting agent Substances 0.000 claims abstract description 6
- 210000004027 cell Anatomy 0.000 claims description 85
- 102000039446 nucleic acids Human genes 0.000 claims description 58
- 108020004707 nucleic acids Proteins 0.000 claims description 58
- 150000007523 nucleic acids Chemical class 0.000 claims description 58
- 238000000034 method Methods 0.000 claims description 37
- 102000004169 proteins and genes Human genes 0.000 claims description 36
- 241000124008 Mammalia Species 0.000 claims description 31
- 230000014509 gene expression Effects 0.000 claims description 31
- 108091033319 polynucleotide Proteins 0.000 claims description 26
- 102000040430 polynucleotide Human genes 0.000 claims description 26
- 239000002157 polynucleotide Substances 0.000 claims description 26
- 210000002569 neuron Anatomy 0.000 claims description 24
- 210000000234 capsid Anatomy 0.000 claims description 18
- 230000002950 deficient Effects 0.000 claims description 12
- 210000004962 mammalian cell Anatomy 0.000 claims description 10
- 210000001789 adipocyte Anatomy 0.000 claims description 9
- 101000617738 Homo sapiens Survival motor neuron protein Proteins 0.000 claims description 3
- 210000000577 adipose tissue Anatomy 0.000 abstract description 28
- 210000003169 central nervous system Anatomy 0.000 abstract description 28
- 239000013607 AAV vector Substances 0.000 abstract description 21
- 210000001519 tissue Anatomy 0.000 abstract description 18
- 230000010415 tropism Effects 0.000 abstract description 14
- 239000008194 pharmaceutical composition Substances 0.000 abstract description 7
- 241000700605 Viruses Species 0.000 description 37
- 108090000565 Capsid Proteins Proteins 0.000 description 29
- 102100023321 Ceruloplasmin Human genes 0.000 description 29
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 25
- 238000010361 transduction Methods 0.000 description 25
- 230000026683 transduction Effects 0.000 description 25
- 108010043121 Green Fluorescent Proteins Proteins 0.000 description 23
- 102000004144 Green Fluorescent Proteins Human genes 0.000 description 23
- 239000005090 green fluorescent protein Substances 0.000 description 23
- 210000004556 brain Anatomy 0.000 description 22
- 108090000765 processed proteins & peptides Proteins 0.000 description 21
- 239000002245 particle Substances 0.000 description 20
- 229920001184 polypeptide Polymers 0.000 description 20
- 102000004196 processed proteins & peptides Human genes 0.000 description 20
- 201000010099 disease Diseases 0.000 description 16
- 239000013608 rAAV vector Substances 0.000 description 16
- 239000000203 mixture Substances 0.000 description 14
- 241001164825 Adeno-associated virus - 8 Species 0.000 description 13
- 208000026911 Tuberous sclerosis complex Diseases 0.000 description 13
- 230000006870 function Effects 0.000 description 12
- 208000002320 spinal muscular atrophy Diseases 0.000 description 12
- 208000026350 Inborn Genetic disease Diseases 0.000 description 11
- 208000016361 genetic disease Diseases 0.000 description 11
- 241001655883 Adeno-associated virus - 1 Species 0.000 description 10
- 238000001415 gene therapy Methods 0.000 description 10
- 238000002347 injection Methods 0.000 description 10
- 239000007924 injection Substances 0.000 description 10
- 108700019146 Transgenes Proteins 0.000 description 9
- 208000035475 disorder Diseases 0.000 description 9
- 238000004519 manufacturing process Methods 0.000 description 9
- 230000004069 differentiation Effects 0.000 description 7
- 239000013604 expression vector Substances 0.000 description 7
- 238000004806 packaging method and process Methods 0.000 description 7
- 230000001225 therapeutic effect Effects 0.000 description 7
- 241000702423 Adeno-associated virus - 2 Species 0.000 description 6
- 101710175981 Hamartin Proteins 0.000 description 6
- 108050009309 Tuberin Proteins 0.000 description 6
- 102000044632 Tuberous Sclerosis Complex 1 Human genes 0.000 description 6
- 230000012010 growth Effects 0.000 description 6
- 230000004048 modification Effects 0.000 description 6
- 238000012986 modification Methods 0.000 description 6
- 239000002773 nucleotide Substances 0.000 description 6
- 125000003729 nucleotide group Chemical group 0.000 description 6
- 230000003612 virological effect Effects 0.000 description 6
- 208000012902 Nervous system disease Diseases 0.000 description 5
- 150000001413 amino acids Chemical group 0.000 description 5
- 210000001130 astrocyte Anatomy 0.000 description 5
- 208000006132 lipodystrophy Diseases 0.000 description 5
- 230000035772 mutation Effects 0.000 description 5
- 210000001577 neostriatum Anatomy 0.000 description 5
- 210000000278 spinal cord Anatomy 0.000 description 5
- 238000013518 transcription Methods 0.000 description 5
- 230000035897 transcription Effects 0.000 description 5
- 102100038369 1-acyl-sn-glycerol-3-phosphate acyltransferase beta Human genes 0.000 description 4
- 208000014644 Brain disease Diseases 0.000 description 4
- 108700028146 Genetic Enhancer Elements Proteins 0.000 description 4
- 102100039289 Glial fibrillary acidic protein Human genes 0.000 description 4
- 101710193519 Glial fibrillary acidic protein Proteins 0.000 description 4
- 101000605571 Homo sapiens 1-acyl-sn-glycerol-3-phosphate acyltransferase beta Proteins 0.000 description 4
- 101000741790 Homo sapiens Peroxisome proliferator-activated receptor gamma Proteins 0.000 description 4
- 101001003584 Homo sapiens Prelamin-A/C Proteins 0.000 description 4
- 101000798015 Homo sapiens RAC-beta serine/threonine-protein kinase Proteins 0.000 description 4
- 101000898985 Homo sapiens Seipin Proteins 0.000 description 4
- 108020004684 Internal Ribosome Entry Sites Proteins 0.000 description 4
- 241000699670 Mus sp. Species 0.000 description 4
- 206010028980 Neoplasm Diseases 0.000 description 4
- 208000025966 Neurological disease Diseases 0.000 description 4
- 102100038825 Peroxisome proliferator-activated receptor gamma Human genes 0.000 description 4
- 102100026531 Prelamin-A/C Human genes 0.000 description 4
- 102100032315 RAC-beta serine/threonine-protein kinase Human genes 0.000 description 4
- 102100021463 Seipin Human genes 0.000 description 4
- 102100021947 Survival motor neuron protein Human genes 0.000 description 4
- 102000002027 Tuberin Human genes 0.000 description 4
- 108091009221 ZMPSTE24 Proteins 0.000 description 4
- 230000009286 beneficial effect Effects 0.000 description 4
- 230000001413 cellular effect Effects 0.000 description 4
- 230000007547 defect Effects 0.000 description 4
- 230000007812 deficiency Effects 0.000 description 4
- 239000003623 enhancer Substances 0.000 description 4
- 239000012634 fragment Substances 0.000 description 4
- 210000005046 glial fibrillary acidic protein Anatomy 0.000 description 4
- 238000000338 in vitro Methods 0.000 description 4
- 239000000463 material Substances 0.000 description 4
- 210000002161 motor neuron Anatomy 0.000 description 4
- 210000002353 nuclear lamina Anatomy 0.000 description 4
- 239000013612 plasmid Substances 0.000 description 4
- 208000024891 symptom Diseases 0.000 description 4
- 210000001103 thalamus Anatomy 0.000 description 4
- 230000002463 transducing effect Effects 0.000 description 4
- 238000013519 translation Methods 0.000 description 4
- 210000002845 virion Anatomy 0.000 description 4
- 241001634120 Adeno-associated virus - 5 Species 0.000 description 3
- 102100031561 Hamartin Human genes 0.000 description 3
- 230000001594 aberrant effect Effects 0.000 description 3
- 210000005013 brain tissue Anatomy 0.000 description 3
- 239000000969 carrier Substances 0.000 description 3
- 238000011161 development Methods 0.000 description 3
- 239000003814 drug Substances 0.000 description 3
- 238000001727 in vivo Methods 0.000 description 3
- 230000000670 limiting effect Effects 0.000 description 3
- 230000002101 lytic effect Effects 0.000 description 3
- 230000008488 polyadenylation Effects 0.000 description 3
- 230000000644 propagated effect Effects 0.000 description 3
- 230000010076 replication Effects 0.000 description 3
- 210000002027 skeletal muscle Anatomy 0.000 description 3
- 230000005100 tissue tropism Effects 0.000 description 3
- 230000005030 transcription termination Effects 0.000 description 3
- 239000013603 viral vector Substances 0.000 description 3
- XLYOFNOQVPJJNP-UHFFFAOYSA-N water Substances O XLYOFNOQVPJJNP-UHFFFAOYSA-N 0.000 description 3
- XKRFYHLGVUSROY-UHFFFAOYSA-N Argon Chemical compound [Ar] XKRFYHLGVUSROY-UHFFFAOYSA-N 0.000 description 2
- 208000006096 Attention Deficit Disorder with Hyperactivity Diseases 0.000 description 2
- 208000036864 Attention deficit/hyperactivity disease Diseases 0.000 description 2
- 206010003805 Autism Diseases 0.000 description 2
- 208000020706 Autistic disease Diseases 0.000 description 2
- 241000283707 Capra Species 0.000 description 2
- 102000005720 Glutathione transferase Human genes 0.000 description 2
- 108010070675 Glutathione transferase Proteins 0.000 description 2
- 241001465754 Metazoa Species 0.000 description 2
- 108700026244 Open Reading Frames Proteins 0.000 description 2
- 241000288906 Primates Species 0.000 description 2
- 238000000692 Student's t-test Methods 0.000 description 2
- 230000010014 adipocyte dysfunction Effects 0.000 description 2
- 239000002671 adjuvant Substances 0.000 description 2
- 230000002411 adverse Effects 0.000 description 2
- 125000000539 amino acid group Chemical group 0.000 description 2
- 210000004436 artificial bacterial chromosome Anatomy 0.000 description 2
- 210000001106 artificial yeast chromosome Anatomy 0.000 description 2
- 230000003140 astrocytic effect Effects 0.000 description 2
- 208000015802 attention deficit-hyperactivity disease Diseases 0.000 description 2
- 230000008499 blood brain barrier function Effects 0.000 description 2
- 210000001218 blood-brain barrier Anatomy 0.000 description 2
- 210000004720 cerebrum Anatomy 0.000 description 2
- 238000004624 confocal microscopy Methods 0.000 description 2
- 238000007796 conventional method Methods 0.000 description 2
- 210000001653 corpus striatum Anatomy 0.000 description 2
- 230000001054 cortical effect Effects 0.000 description 2
- 229940079593 drug Drugs 0.000 description 2
- 239000003937 drug carrier Substances 0.000 description 2
- 239000000839 emulsion Substances 0.000 description 2
- 238000009472 formulation Methods 0.000 description 2
- 210000001905 globus pallidus Anatomy 0.000 description 2
- 210000001320 hippocampus Anatomy 0.000 description 2
- 238000003364 immunohistochemistry Methods 0.000 description 2
- 230000001976 improved effect Effects 0.000 description 2
- 230000001939 inductive effect Effects 0.000 description 2
- 230000002401 inhibitory effect Effects 0.000 description 2
- 210000004185 liver Anatomy 0.000 description 2
- 230000033001 locomotion Effects 0.000 description 2
- 230000001404 mediated effect Effects 0.000 description 2
- 210000003205 muscle Anatomy 0.000 description 2
- 201000006938 muscular dystrophy Diseases 0.000 description 2
- 210000004165 myocardium Anatomy 0.000 description 2
- 210000005036 nerve Anatomy 0.000 description 2
- 210000000653 nervous system Anatomy 0.000 description 2
- 230000001537 neural effect Effects 0.000 description 2
- 210000004498 neuroglial cell Anatomy 0.000 description 2
- 238000001543 one-way ANOVA Methods 0.000 description 2
- 210000000056 organ Anatomy 0.000 description 2
- 230000035699 permeability Effects 0.000 description 2
- -1 phage Substances 0.000 description 2
- 239000002953 phosphate buffered saline Substances 0.000 description 2
- 238000002360 preparation method Methods 0.000 description 2
- 238000003753 real-time PCR Methods 0.000 description 2
- 230000001603 reducing effect Effects 0.000 description 2
- 238000011160 research Methods 0.000 description 2
- 238000007619 statistical method Methods 0.000 description 2
- 210000003523 substantia nigra Anatomy 0.000 description 2
- 238000012360 testing method Methods 0.000 description 2
- 230000000542 thalamic effect Effects 0.000 description 2
- 230000000699 topical effect Effects 0.000 description 2
- SZCZSKMCTGEJKI-UHFFFAOYSA-N tuberin Natural products COC1=CC=C(C=CNC=O)C=C1 SZCZSKMCTGEJKI-UHFFFAOYSA-N 0.000 description 2
- 238000002604 ultrasonography Methods 0.000 description 2
- 241000701161 unidentified adenovirus Species 0.000 description 2
- 102000007469 Actins Human genes 0.000 description 1
- 108010085238 Actins Proteins 0.000 description 1
- 241000972680 Adeno-associated virus - 6 Species 0.000 description 1
- 208000024827 Alzheimer disease Diseases 0.000 description 1
- 241000224489 Amoeba Species 0.000 description 1
- 206010002091 Anaesthesia Diseases 0.000 description 1
- 206010002329 Aneurysm Diseases 0.000 description 1
- 208000035143 Bacterial infection Diseases 0.000 description 1
- 102100022548 Beta-hexosaminidase subunit alpha Human genes 0.000 description 1
- 208000003174 Brain Neoplasms Diseases 0.000 description 1
- 238000011740 C57BL/6 mouse Methods 0.000 description 1
- 208000022526 Canavan disease Diseases 0.000 description 1
- 102100022641 Coagulation factor IX Human genes 0.000 description 1
- 208000003322 Coinfection Diseases 0.000 description 1
- 206010010071 Coma Diseases 0.000 description 1
- 206010010904 Convulsion Diseases 0.000 description 1
- 201000003883 Cystic fibrosis Diseases 0.000 description 1
- 108020004414 DNA Proteins 0.000 description 1
- 102000053602 DNA Human genes 0.000 description 1
- 206010012289 Dementia Diseases 0.000 description 1
- 201000010374 Down Syndrome Diseases 0.000 description 1
- 208000032274 Encephalopathy Diseases 0.000 description 1
- 241000283074 Equus asinus Species 0.000 description 1
- 201000004311 Gilles de la Tourette syndrome Diseases 0.000 description 1
- KOSRFJWDECSPRO-WDSKDSINSA-N Glu-Glu Chemical compound OC(=O)CC[C@H](N)C(=O)N[C@@H](CCC(O)=O)C(O)=O KOSRFJWDECSPRO-WDSKDSINSA-N 0.000 description 1
- 229940033330 HIV vaccine Drugs 0.000 description 1
- 208000023105 Huntington disease Diseases 0.000 description 1
- 206010062767 Hypophysitis Diseases 0.000 description 1
- 206010061218 Inflammation Diseases 0.000 description 1
- 201000006347 Intellectual Disability Diseases 0.000 description 1
- YQEZLKZALYSWHR-UHFFFAOYSA-N Ketamine Chemical compound C=1C=CC=C(Cl)C=1C1(NC)CCCCC1=O YQEZLKZALYSWHR-UHFFFAOYSA-N 0.000 description 1
- 241000282567 Macaca fascicularis Species 0.000 description 1
- 201000009906 Meningitis Diseases 0.000 description 1
- 208000026072 Motor neurone disease Diseases 0.000 description 1
- 241000699666 Mus <mouse, genus> Species 0.000 description 1
- 241000283973 Oryctolagus cuniculus Species 0.000 description 1
- 206010033799 Paralysis Diseases 0.000 description 1
- 208000030852 Parasitic disease Diseases 0.000 description 1
- 208000018737 Parkinson disease Diseases 0.000 description 1
- 241000701945 Parvoviridae Species 0.000 description 1
- QGMRQYFBGABWDR-UHFFFAOYSA-M Pentobarbital sodium Chemical compound [Na+].CCCC(C)C1(CC)C(=O)NC(=O)[N-]C1=O QGMRQYFBGABWDR-UHFFFAOYSA-M 0.000 description 1
- 108010076504 Protein Sorting Signals Proteins 0.000 description 1
- 206010037742 Rabies Diseases 0.000 description 1
- 208000005793 Restless legs syndrome Diseases 0.000 description 1
- 101150081851 SMN1 gene Proteins 0.000 description 1
- 241000700584 Simplexvirus Species 0.000 description 1
- 108020004682 Single-Stranded DNA Proteins 0.000 description 1
- 108091081024 Start codon Proteins 0.000 description 1
- 108010090804 Streptavidin Proteins 0.000 description 1
- 208000006011 Stroke Diseases 0.000 description 1
- CZMRCDWAGMRECN-UGDNZRGBSA-N Sucrose Chemical compound O[C@H]1[C@H](O)[C@@H](CO)O[C@@]1(CO)O[C@@H]1[C@H](O)[C@@H](O)[C@H](O)[C@@H](CO)O1 CZMRCDWAGMRECN-UGDNZRGBSA-N 0.000 description 1
- 229930006000 Sucrose Natural products 0.000 description 1
- 208000022292 Tay-Sachs disease Diseases 0.000 description 1
- 208000026062 Tissue disease Diseases 0.000 description 1
- 208000000323 Tourette Syndrome Diseases 0.000 description 1
- 208000016620 Tourette disease Diseases 0.000 description 1
- 206010044565 Tremor Diseases 0.000 description 1
- 229920004890 Triton X-100 Polymers 0.000 description 1
- 239000013504 Triton X-100 Substances 0.000 description 1
- 208000036142 Viral infection Diseases 0.000 description 1
- 241001492404 Woodchuck hepatitis virus Species 0.000 description 1
- 239000004480 active ingredient Substances 0.000 description 1
- 210000003486 adipose tissue brown Anatomy 0.000 description 1
- 210000000593 adipose tissue white Anatomy 0.000 description 1
- 239000000443 aerosol Substances 0.000 description 1
- 230000016571 aggressive behavior Effects 0.000 description 1
- KOSRFJWDECSPRO-UHFFFAOYSA-N alpha-L-glutamyl-L-glutamic acid Natural products OC(=O)CCC(N)C(=O)NC(CCC(O)=O)C(O)=O KOSRFJWDECSPRO-UHFFFAOYSA-N 0.000 description 1
- 210000004727 amygdala Anatomy 0.000 description 1
- 238000001949 anaesthesia Methods 0.000 description 1
- 230000037005 anaesthesia Effects 0.000 description 1
- 238000000540 analysis of variance Methods 0.000 description 1
- 238000010171 animal model Methods 0.000 description 1
- 230000000692 anti-sense effect Effects 0.000 description 1
- 230000000890 antigenic effect Effects 0.000 description 1
- 229910052786 argon Inorganic materials 0.000 description 1
- 230000003190 augmentative effect Effects 0.000 description 1
- 210000000467 autonomic pathway Anatomy 0.000 description 1
- 208000022362 bacterial infectious disease Diseases 0.000 description 1
- 210000004227 basal ganglia Anatomy 0.000 description 1
- 231100000871 behavioral problem Toxicity 0.000 description 1
- 230000008901 benefit Effects 0.000 description 1
- 208000013355 benign neoplasm of brain Diseases 0.000 description 1
- 102000023732 binding proteins Human genes 0.000 description 1
- 108091008324 binding proteins Proteins 0.000 description 1
- 230000004071 biological effect Effects 0.000 description 1
- 230000000903 blocking effect Effects 0.000 description 1
- 108010006025 bovine growth hormone Proteins 0.000 description 1
- 208000029028 brain injury Diseases 0.000 description 1
- 210000000133 brain stem Anatomy 0.000 description 1
- 239000000872 buffer Substances 0.000 description 1
- 201000011510 cancer Diseases 0.000 description 1
- 239000013592 cell lysate Substances 0.000 description 1
- 210000001638 cerebellum Anatomy 0.000 description 1
- 206010008129 cerebral palsy Diseases 0.000 description 1
- 210000001175 cerebrospinal fluid Anatomy 0.000 description 1
- 239000003153 chemical reaction reagent Substances 0.000 description 1
- 239000003795 chemical substances by application Substances 0.000 description 1
- 210000000349 chromosome Anatomy 0.000 description 1
- 208000037976 chronic inflammation Diseases 0.000 description 1
- 230000006020 chronic inflammation Effects 0.000 description 1
- 230000008045 co-localization Effects 0.000 description 1
- 210000000860 cochlear nerve Anatomy 0.000 description 1
- 230000001149 cognitive effect Effects 0.000 description 1
- 230000000295 complement effect Effects 0.000 description 1
- 150000001875 compounds Chemical class 0.000 description 1
- 238000011109 contamination Methods 0.000 description 1
- 230000001276 controlling effect Effects 0.000 description 1
- 230000003247 decreasing effect Effects 0.000 description 1
- 238000012217 deletion Methods 0.000 description 1
- 230000037430 deletion Effects 0.000 description 1
- 238000001514 detection method Methods 0.000 description 1
- 210000000188 diaphragm Anatomy 0.000 description 1
- 238000009792 diffusion process Methods 0.000 description 1
- LOKCTEFSRHRXRJ-UHFFFAOYSA-I dipotassium trisodium dihydrogen phosphate hydrogen phosphate dichloride Chemical compound P(=O)(O)(O)[O-].[K+].P(=O)(O)([O-])[O-].[Na+].[Na+].[Cl-].[K+].[Cl-].[Na+] LOKCTEFSRHRXRJ-UHFFFAOYSA-I 0.000 description 1
- 210000005110 dorsal hippocampus Anatomy 0.000 description 1
- 239000002552 dosage form Substances 0.000 description 1
- 238000012377 drug delivery Methods 0.000 description 1
- 230000004064 dysfunction Effects 0.000 description 1
- 230000000694 effects Effects 0.000 description 1
- 230000002708 enhancing effect Effects 0.000 description 1
- 206010015037 epilepsy Diseases 0.000 description 1
- 210000000647 epithalamus Anatomy 0.000 description 1
- 239000013613 expression plasmid Substances 0.000 description 1
- 239000000835 fiber Substances 0.000 description 1
- 210000001652 frontal lobe Anatomy 0.000 description 1
- 210000000609 ganglia Anatomy 0.000 description 1
- 238000001476 gene delivery Methods 0.000 description 1
- 230000002068 genetic effect Effects 0.000 description 1
- 230000002518 glial effect Effects 0.000 description 1
- 108010055341 glutamyl-glutamic acid Proteins 0.000 description 1
- 230000036541 health Effects 0.000 description 1
- 208000009429 hemophilia B Diseases 0.000 description 1
- 208000013403 hyperactivity Diseases 0.000 description 1
- 210000003016 hypothalamus Anatomy 0.000 description 1
- 238000007654 immersion Methods 0.000 description 1
- 230000008105 immune reaction Effects 0.000 description 1
- 230000028993 immune response Effects 0.000 description 1
- 208000035231 inattentive type attention deficit hyperactivity disease Diseases 0.000 description 1
- 238000010348 incorporation Methods 0.000 description 1
- 208000015181 infectious disease Diseases 0.000 description 1
- 210000003552 inferior colliculi Anatomy 0.000 description 1
- 230000004054 inflammatory process Effects 0.000 description 1
- 239000004615 ingredient Substances 0.000 description 1
- 230000000977 initiatory effect Effects 0.000 description 1
- 238000003780 insertion Methods 0.000 description 1
- 230000037431 insertion Effects 0.000 description 1
- 238000007918 intramuscular administration Methods 0.000 description 1
- 238000007913 intrathecal administration Methods 0.000 description 1
- 238000001990 intravenous administration Methods 0.000 description 1
- NBQNWMBBSKPBAY-UHFFFAOYSA-N iodixanol Chemical compound IC=1C(C(=O)NCC(O)CO)=C(I)C(C(=O)NCC(O)CO)=C(I)C=1N(C(=O)C)CC(O)CN(C(C)=O)C1=C(I)C(C(=O)NCC(O)CO)=C(I)C(C(=O)NCC(O)CO)=C1I NBQNWMBBSKPBAY-UHFFFAOYSA-N 0.000 description 1
- 229960004359 iodixanol Drugs 0.000 description 1
- 229960003299 ketamine Drugs 0.000 description 1
- 210000003734 kidney Anatomy 0.000 description 1
- 210000003715 limbic system Anatomy 0.000 description 1
- 238000009593 lumbar puncture Methods 0.000 description 1
- 210000004072 lung Anatomy 0.000 description 1
- 210000001165 lymph node Anatomy 0.000 description 1
- 230000036244 malformation Effects 0.000 description 1
- 239000003550 marker Substances 0.000 description 1
- 238000005259 measurement Methods 0.000 description 1
- 210000001767 medulla oblongata Anatomy 0.000 description 1
- 201000001441 melanoma Diseases 0.000 description 1
- 210000001259 mesencephalon Anatomy 0.000 description 1
- 210000000274 microglia Anatomy 0.000 description 1
- 239000002991 molded plastic Substances 0.000 description 1
- 208000005264 motor neuron disease Diseases 0.000 description 1
- 239000012120 mounting media Substances 0.000 description 1
- 201000006417 multiple sclerosis Diseases 0.000 description 1
- 206010028417 myasthenia gravis Diseases 0.000 description 1
- 201000003631 narcolepsy Diseases 0.000 description 1
- 210000000478 neocortex Anatomy 0.000 description 1
- 210000003061 neural cell Anatomy 0.000 description 1
- 208000015122 neurodegenerative disease Diseases 0.000 description 1
- 230000000926 neurological effect Effects 0.000 description 1
- 230000006764 neuronal dysfunction Effects 0.000 description 1
- 201000001119 neuropathy Diseases 0.000 description 1
- 230000007823 neuropathy Effects 0.000 description 1
- 230000002276 neurotropic effect Effects 0.000 description 1
- 210000000869 occipital lobe Anatomy 0.000 description 1
- 210000004248 oligodendroglia Anatomy 0.000 description 1
- 230000036281 parasite infection Effects 0.000 description 1
- 230000001936 parietal effect Effects 0.000 description 1
- 210000001152 parietal lobe Anatomy 0.000 description 1
- 230000036961 partial effect Effects 0.000 description 1
- 230000007170 pathology Effects 0.000 description 1
- 229940124531 pharmaceutical excipient Drugs 0.000 description 1
- 210000004560 pineal gland Anatomy 0.000 description 1
- 210000003635 pituitary gland Anatomy 0.000 description 1
- 229920002704 polyhistidine Polymers 0.000 description 1
- 230000001124 posttranscriptional effect Effects 0.000 description 1
- 210000002307 prostate Anatomy 0.000 description 1
- 238000000746 purification Methods 0.000 description 1
- 238000011084 recovery Methods 0.000 description 1
- 230000009467 reduction Effects 0.000 description 1
- 230000011514 reflex Effects 0.000 description 1
- 230000001105 regulatory effect Effects 0.000 description 1
- 230000004044 response Effects 0.000 description 1
- 230000000717 retained effect Effects 0.000 description 1
- 210000001525 retina Anatomy 0.000 description 1
- 230000002207 retinal effect Effects 0.000 description 1
- 230000007441 retrograde transport Effects 0.000 description 1
- 206010039073 rheumatoid arthritis Diseases 0.000 description 1
- 230000001953 sensory effect Effects 0.000 description 1
- 210000002966 serum Anatomy 0.000 description 1
- 239000000243 solution Substances 0.000 description 1
- 238000010245 stereological analysis Methods 0.000 description 1
- 238000007920 subcutaneous administration Methods 0.000 description 1
- 239000005720 sucrose Substances 0.000 description 1
- 238000001356 surgical procedure Methods 0.000 description 1
- 230000008685 targeting Effects 0.000 description 1
- 210000001587 telencephalon Anatomy 0.000 description 1
- 230000002123 temporal effect Effects 0.000 description 1
- 210000003478 temporal lobe Anatomy 0.000 description 1
- MPLHNVLQVRSVEE-UHFFFAOYSA-N texas red Chemical compound [O-]S(=O)(=O)C1=CC(S(Cl)(=O)=O)=CC=C1C(C1=CC=2CCCN3CCCC(C=23)=C1O1)=C2C1=C(CCC1)C3=[N+]1CCCC3=C2 MPLHNVLQVRSVEE-UHFFFAOYSA-N 0.000 description 1
- 238000002560 therapeutic procedure Methods 0.000 description 1
- 238000001890 transfection Methods 0.000 description 1
- 238000012546 transfer Methods 0.000 description 1
- 238000005199 ultracentrifugation Methods 0.000 description 1
- 241000701447 unidentified baculovirus Species 0.000 description 1
- 241001529453 unidentified herpesvirus Species 0.000 description 1
- 210000004291 uterus Anatomy 0.000 description 1
- 210000005111 ventral hippocampus Anatomy 0.000 description 1
- 230000029812 viral genome replication Effects 0.000 description 1
- 230000009385 viral infection Effects 0.000 description 1
- 239000000080 wetting agent Substances 0.000 description 1
- BPICBUSOMSTKRF-UHFFFAOYSA-N xylazine Chemical compound CC1=CC=CC(C)=C1NC1=NCCCS1 BPICBUSOMSTKRF-UHFFFAOYSA-N 0.000 description 1
- 229960001600 xylazine Drugs 0.000 description 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
- 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
-
- 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
- C12N15/86—Viral vectors
-
- 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
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
-
- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- 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
- A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762550458P | 2017-08-25 | 2017-08-25 | |
| US62/550,458 | 2017-08-25 | ||
| PCT/US2018/047466 WO2019040586A1 (en) | 2017-08-25 | 2018-08-22 | RECOMBINANT ADENO-ASSOCIATE VECTORS |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AU2018320849A1 true AU2018320849A1 (en) | 2020-03-05 |
Family
ID=65439655
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2018320849A Abandoned AU2018320849A1 (en) | 2017-08-25 | 2018-08-22 | Recombinant adeno-associated vectors |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US20190085358A1 (enExample) |
| EP (1) | EP3655041A4 (enExample) |
| JP (1) | JP2020533968A (enExample) |
| KR (1) | KR20200042935A (enExample) |
| CN (1) | CN111163811A (enExample) |
| AU (1) | AU2018320849A1 (enExample) |
| CA (1) | CA3073937A1 (enExample) |
| IL (1) | IL272655A (enExample) |
| MX (1) | MX2020002148A (enExample) |
| WO (1) | WO2019040586A1 (enExample) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN113395962A (zh) | 2018-11-21 | 2021-09-14 | Certego治疗公司 | 加波沙朵用于降低自杀风险和快速缓解抑郁症 |
| CA3149164A1 (en) * | 2019-09-03 | 2021-03-11 | Suma Krishnan | Compositions and methods for the treatment of congenital ichthyoses |
| AU2021275863A1 (en) | 2020-05-20 | 2023-02-02 | Certego Therapeutics Inc. | Ring deuterated gaboxadol and its use for the treatment of psychiatric disorders |
| CA3191543A1 (en) * | 2020-09-04 | 2022-03-10 | Lei Cao | Novel engineered capsid serotype of recombinant adeno-associated viral vector with enhanced transduction efficiency and widespread distribution in the brain |
| US20230086710A1 (en) * | 2021-09-14 | 2023-03-23 | California Institute Of Technology | Aav capsid variants |
Family Cites Families (31)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO1999061601A2 (en) * | 1998-05-28 | 1999-12-02 | The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Aav5 vector and uses thereof |
| JP4969002B2 (ja) * | 1999-06-08 | 2012-07-04 | ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン | ｒＡＡＶ形質導入を増加するための化合物および方法 |
| AU2003259803B2 (en) * | 2002-08-12 | 2007-08-02 | The Regents Of The University Of Michigan | Diagnosis and treatment of tuberous sclerosis |
| US7427396B2 (en) * | 2004-06-03 | 2008-09-23 | Genzyme Corporation | AAV vectors for gene delivery to the lung |
| US7838657B2 (en) * | 2004-12-03 | 2010-11-23 | University Of Massachusetts | Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences |
| EP3409296A1 (en) * | 2005-04-07 | 2018-12-05 | The Trustees of the University of Pennsylvania | Method of increasing the function of an aav vector |
| US9415121B2 (en) * | 2008-12-19 | 2016-08-16 | Nationwide Children's Hospital | Delivery of MECP2 polynucleotide using recombinant AAV9 |
| EP2692868A1 (en) * | 2012-08-02 | 2014-02-05 | Universitat Autònoma De Barcelona | Adeno-associated viral (AAV) vectors useful for transducing adipose tissue |
| AU2013388083B2 (en) * | 2013-05-01 | 2019-08-22 | Genzyme Corporation | Compositions and methods for treating spinal muscular atrophy |
| PH12016500162B1 (en) * | 2013-07-22 | 2024-02-21 | Childrens Hospital Philadelphia | Variant aav and compositions, methods and uses for gene trnsfer to cells, organs, and tissues |
| GB201403684D0 (en) * | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| WO2015171547A1 (en) * | 2014-05-05 | 2015-11-12 | Ovid Therapeutics Inc. | Methods of treating cognitive impairment associated with neurodegenerative disorders |
| AU2015266770A1 (en) * | 2014-05-30 | 2016-12-08 | The Board Of Trustees Of The Leland Stanford Junior University | Compositions and methods of delivering treatments for latent viral infections |
| EP3151866B1 (en) * | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
| JP6842410B2 (ja) * | 2014-10-03 | 2021-03-17 | ユニバーシティ オブ マサチューセッツ | 新規の高効率ライブラリーにより同定されるａａｖベクター |
| WO2017100671A1 (en) * | 2015-12-11 | 2017-06-15 | California Institute Of Technology | TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs) |
| WO2017106354A1 (en) * | 2015-12-14 | 2017-06-22 | The Trustees Of The University Of Pennsylvania | Adeno-associated viral vectors useful in treatment of spinal muscular atropy |
| MX2018013463A (es) * | 2016-05-13 | 2019-07-04 | 4D Molecular Therapeutics Inc | Capsides variantes de virus adenoasociados y metodos de uso de las mismas. |
| IL268534B2 (en) * | 2017-02-15 | 2024-10-01 | Univ North Carolina Chapel Hill | Methods and compositions for gene transfer across the vasculature |
| EP3595669A4 (en) * | 2017-03-15 | 2021-03-03 | Ovid Therapeutics Inc. | USE OF TAILOR-MADE RECEPTORS ACTIVATED EXCLUSIVELY BY CUSTOM-MADE MEDICINES IN THE TREATMENT OF EPILEPTIC DISORDERS |
| JP2021506908A (ja) * | 2017-12-20 | 2021-02-22 | オービッド・セラピューティクス・インコーポレイテッドＯｖｉｄ Ｔｈｅｒａｐｅｕｔｉｃｓ Ｉｎｃ． | 発作障害の治療におけるｈＭ４Ｄｉの使用 |
| US10704048B2 (en) * | 2018-06-14 | 2020-07-07 | Ovid Therapeutics Inc. | Use of MIR-92A or MIR-145 in the treatment of Angelman syndrome |
| EP3856913A4 (en) * | 2018-09-26 | 2022-10-26 | California Institute Of Technology | Adeno-associated virus compositions for targeted gene therapy |
| US10557149B1 (en) * | 2019-07-15 | 2020-02-11 | Vigene Biosciences, Inc. | Recombinantly-modified adeno-associated virus helper vectors and their use to improve the packaging efficiency of recombinantly-modified adeno-associated virus |
| PH12022551229A1 (en) * | 2019-11-19 | 2023-07-31 | Asklepios Biopharmaceutical Inc | Therapeutic adeno-associated virus comprising liver-specific promoters for treating pompe disease and lysosomal disorders |
| JP7702949B2 (ja) * | 2019-12-05 | 2025-07-04 | ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム | ヒトｍｅｃｐ２遺伝子を発現するように設計されたトランスジーンカセット |
| CA3116391A1 (en) * | 2020-09-14 | 2022-03-14 | President And Fellows Of Harvard College | Recombinant adeno associated virus encoding clarin-1 and uses thereof |
| EP4288445A1 (en) * | 2021-02-03 | 2023-12-13 | The University of North Carolina at Chapel Hill | Protein m analogs and fusion proteins and their use for inhibiting antibody function |
| WO2022235702A1 (en) * | 2021-05-04 | 2022-11-10 | California Institute Of Technology | Recombinant aavs for delivery to central nervous system and brain vasculature |
| CN114259502A (zh) * | 2021-12-24 | 2022-04-01 | 南京鼓楼医院 | 一种基于脂肪组织的中枢靶向递送siRNA的方法及应用 |
| CN115554418B (zh) * | 2022-11-22 | 2023-04-14 | 四川至善唯新生物科技有限公司 | 一种重组腺相关病毒载体的药物组合物及其用途 |
-
2018
- 2018-08-22 CN CN201880064381.6A patent/CN111163811A/zh active Pending
- 2018-08-22 JP JP2020511199A patent/JP2020533968A/ja active Pending
- 2018-08-22 MX MX2020002148A patent/MX2020002148A/es unknown
- 2018-08-22 US US16/108,393 patent/US20190085358A1/en not_active Abandoned
- 2018-08-22 CA CA3073937A patent/CA3073937A1/en active Pending
- 2018-08-22 KR KR1020207008689A patent/KR20200042935A/ko not_active Withdrawn
- 2018-08-22 WO PCT/US2018/047466 patent/WO2019040586A1/en not_active Ceased
- 2018-08-22 EP EP18849356.3A patent/EP3655041A4/en not_active Withdrawn
- 2018-08-22 AU AU2018320849A patent/AU2018320849A1/en not_active Abandoned
-
2020
- 2020-02-13 IL IL272655A patent/IL272655A/en unknown
-
2021
- 2021-08-12 US US17/400,768 patent/US20210371880A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| IL272655A (en) | 2020-03-31 |
| US20210371880A1 (en) | 2021-12-02 |
| MX2020002148A (es) | 2020-07-20 |
| EP3655041A4 (en) | 2021-04-21 |
| WO2019040586A1 (en) | 2019-02-28 |
| JP2020533968A (ja) | 2020-11-26 |
| KR20200042935A (ko) | 2020-04-24 |
| EP3655041A1 (en) | 2020-05-27 |
| US20190085358A1 (en) | 2019-03-21 |
| CN111163811A (zh) | 2020-05-15 |
| CA3073937A1 (en) | 2019-02-28 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US11802293B2 (en) | Adeno-associated virus vector | |
| US20210371880A1 (en) | Recombinant adeno-associated vectors | |
| ES2599632T3 (es) | Composiciones de vectores rAAV que tienen proteínas de la cápside modificadas en tirosina y métodos para su uso | |
| JP2019216724A (ja) | Ａａｖ５カプシドタンパク質を含むアデノ随伴ウイルス（ａａｖ）を用いた神経学的疾患の処置 | |
| CN103189507A (zh) | 用于向神经系统细胞导入基因的腺相关病毒粒子 | |
| RU2751592C2 (ru) | Выделенный модифицированный белок VP1 капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе | |
| US20250011810A1 (en) | Recombinant aav formulations | |
| CN114126667A (zh) | 使用糖苷水解酶的基因疗法载体向视网膜细胞的改进递送 | |
| JP7781057B2 (ja) | Ｉｒｆ２ｂｐｌ遺伝子の変異に関連する障害の治療のための材料および方法 | |
| JP2024515902A (ja) | 組織向性が改善されたアデノ随伴ウイルスベクターカプシド | |
| JP2023536067A (ja) | 最適化されたｓｌｃ１３ａ５遺伝子および発現カセットならびにそれらの使用 | |
| RU2825667C2 (ru) | Выделенный модифицированный белок VPI капсида аденоассоциированного вируса 9 серотипа (AAV9), капсид и вектор на его основе | |
| EA048714B1 (ru) | Выделенный модифицированный белок vp1 капсида аденоассоциированного вируса 9 серотипа (aav9), капсид и вектор на его основе | |
| CN119894920A (zh) | 用于靶向神经系统的新型aav衣壳及其用途 | |
| EA045824B1 (ru) | Выделенный модифицированный белок vp1 капсида aav5 | |
| HK40019369A (en) | Adeno-associated virus vector |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| MK4 | Application lapsed section 142(2)(d) - no continuation fee paid for the application |