DK2822964T3 - IDENTIFICATION OF CHANNEL RHODOPSIN-2- (CHOP2) MUTATIONS AND METHODS OF USE - Google Patents
IDENTIFICATION OF CHANNEL RHODOPSIN-2- (CHOP2) MUTATIONS AND METHODS OF USE Download PDFInfo
- Publication number
- DK2822964T3 DK2822964T3 DK13710253.9T DK13710253T DK2822964T3 DK 2822964 T3 DK2822964 T3 DK 2822964T3 DK 13710253 T DK13710253 T DK 13710253T DK 2822964 T3 DK2822964 T3 DK 2822964T3
- Authority
- DK
- Denmark
- Prior art keywords
- cell
- chop2
- cells
- light
- seq
- Prior art date
Links
- 238000000034 method Methods 0.000 title claims description 31
- 230000035772 mutation Effects 0.000 title description 19
- 210000004027 cell Anatomy 0.000 claims description 126
- 235000001014 amino acid Nutrition 0.000 claims description 77
- 229920001184 polypeptide Polymers 0.000 claims description 62
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 62
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 62
- 230000004438 eyesight Effects 0.000 claims description 47
- 210000003994 retinal ganglion cell Anatomy 0.000 claims description 46
- 150000007523 nucleic acids Chemical class 0.000 claims description 32
- 108020004707 nucleic acids Proteins 0.000 claims description 30
- 102000039446 nucleic acids Human genes 0.000 claims description 30
- 206010034960 Photophobia Diseases 0.000 claims description 27
- 208000013469 light sensitivity Diseases 0.000 claims description 27
- 239000000203 mixture Substances 0.000 claims description 27
- 210000000411 amacrine cell Anatomy 0.000 claims description 25
- 150000001413 amino acids Chemical group 0.000 claims description 23
- 235000018417 cysteine Nutrition 0.000 claims description 17
- XUJNEKJLAYXESH-UHFFFAOYSA-N cysteine Natural products SCC(N)C(O)=O XUJNEKJLAYXESH-UHFFFAOYSA-N 0.000 claims description 17
- 108091008695 photoreceptors Proteins 0.000 claims description 17
- 230000000763 evoking effect Effects 0.000 claims description 14
- 239000007924 injection Substances 0.000 claims description 13
- 238000002347 injection Methods 0.000 claims description 13
- 210000002287 horizontal cell Anatomy 0.000 claims description 12
- 208000007014 Retinitis pigmentosa Diseases 0.000 claims description 8
- 210000000857 visual cortex Anatomy 0.000 claims description 8
- MTCFGRXMJLQNBG-UHFFFAOYSA-N Serine Natural products OCC(N)C(O)=O MTCFGRXMJLQNBG-UHFFFAOYSA-N 0.000 claims description 7
- 230000001965 increasing effect Effects 0.000 claims description 7
- 239000003937 drug carrier Substances 0.000 claims description 5
- 230000001771 impaired effect Effects 0.000 claims description 5
- 238000000338 in vitro Methods 0.000 claims description 5
- 208000002780 macular degeneration Diseases 0.000 claims description 4
- 208000030533 eye disease Diseases 0.000 claims 2
- 125000003275 alpha amino acid group Chemical group 0.000 description 68
- 108090000623 proteins and genes Proteins 0.000 description 49
- 230000000007 visual effect Effects 0.000 description 41
- 102000004169 proteins and genes Human genes 0.000 description 37
- 235000018102 proteins Nutrition 0.000 description 36
- 108091006146 Channels Proteins 0.000 description 34
- 210000001525 retina Anatomy 0.000 description 32
- 230000014509 gene expression Effects 0.000 description 25
- NCYCYZXNIZJOKI-UHFFFAOYSA-N vitamin A aldehyde Natural products O=CC=C(C)C=CC=C(C)C=CC1=C(C)CCCC1(C)C NCYCYZXNIZJOKI-UHFFFAOYSA-N 0.000 description 25
- 230000002207 retinal effect Effects 0.000 description 24
- 230000004044 response Effects 0.000 description 23
- 210000000608 photoreceptor cell Anatomy 0.000 description 16
- 238000012360 testing method Methods 0.000 description 16
- ROHFNLRQFUQHCH-YFKPBYRVSA-N L-leucine Chemical compound CC(C)C[C@H](N)C(O)=O ROHFNLRQFUQHCH-YFKPBYRVSA-N 0.000 description 15
- ROHFNLRQFUQHCH-UHFFFAOYSA-N Leucine Natural products CC(C)CC(N)C(O)=O ROHFNLRQFUQHCH-UHFFFAOYSA-N 0.000 description 15
- AYFVYJQAPQTCCC-UHFFFAOYSA-N Threonine Natural products CC(O)C(N)C(O)=O AYFVYJQAPQTCCC-UHFFFAOYSA-N 0.000 description 15
- 239000004473 Threonine Substances 0.000 description 15
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 15
- 230000000694 effects Effects 0.000 description 14
- 238000012421 spiking Methods 0.000 description 14
- 208000003098 Ganglion Cysts Diseases 0.000 description 13
- 241000699670 Mus sp. Species 0.000 description 13
- 208000005400 Synovial Cyst Diseases 0.000 description 13
- 210000001508 eye Anatomy 0.000 description 13
- 230000037361 pathway Effects 0.000 description 12
- 210000002508 rod bipolar cell Anatomy 0.000 description 12
- 201000004569 Blindness Diseases 0.000 description 11
- 208000022873 Ocular disease Diseases 0.000 description 11
- 230000001404 mediated effect Effects 0.000 description 11
- 210000001116 retinal neuron Anatomy 0.000 description 11
- QNAYBMKLOCPYGJ-REOHCLBHSA-N L-alanine Chemical compound C[C@H](N)C(O)=O QNAYBMKLOCPYGJ-REOHCLBHSA-N 0.000 description 10
- 235000004279 alanine Nutrition 0.000 description 10
- 208000035475 disorder Diseases 0.000 description 9
- 230000007935 neutral effect Effects 0.000 description 8
- 210000000225 synapse Anatomy 0.000 description 8
- 238000012549 training Methods 0.000 description 8
- 241000195597 Chlamydomonas reinhardtii Species 0.000 description 7
- 108050001704 Opsin Proteins 0.000 description 7
- 102000010175 Opsin Human genes 0.000 description 6
- 210000004556 brain Anatomy 0.000 description 6
- 230000007850 degeneration Effects 0.000 description 6
- 201000010099 disease Diseases 0.000 description 6
- 230000006870 function Effects 0.000 description 6
- 238000005286 illumination Methods 0.000 description 6
- 230000028161 membrane depolarization Effects 0.000 description 6
- 210000002569 neuron Anatomy 0.000 description 6
- 210000000964 retinal cone photoreceptor cell Anatomy 0.000 description 6
- 230000000638 stimulation Effects 0.000 description 6
- 230000002123 temporal effect Effects 0.000 description 6
- 239000013598 vector Substances 0.000 description 6
- 241001465754 Metazoa Species 0.000 description 5
- 230000004913 activation Effects 0.000 description 5
- 230000009849 deactivation Effects 0.000 description 5
- 238000001727 in vivo Methods 0.000 description 5
- 230000002458 infectious effect Effects 0.000 description 5
- 210000000880 retinal rod photoreceptor cell Anatomy 0.000 description 5
- -1 Ca2+ ions Chemical class 0.000 description 4
- 108020004414 DNA Proteins 0.000 description 4
- 108700008625 Reporter Genes Proteins 0.000 description 4
- 102100040756 Rhodopsin Human genes 0.000 description 4
- 108700019146 Transgenes Proteins 0.000 description 4
- 241000700605 Viruses Species 0.000 description 4
- 238000004458 analytical method Methods 0.000 description 4
- 230000002238 attenuated effect Effects 0.000 description 4
- 239000012634 fragment Substances 0.000 description 4
- 238000001415 gene therapy Methods 0.000 description 4
- 230000002068 genetic effect Effects 0.000 description 4
- 239000007943 implant Substances 0.000 description 4
- 208000015181 infectious disease Diseases 0.000 description 4
- 230000007774 longterm Effects 0.000 description 4
- 108020004999 messenger RNA Proteins 0.000 description 4
- 239000002773 nucleotide Substances 0.000 description 4
- 125000003729 nucleotide group Chemical group 0.000 description 4
- 108091033319 polynucleotide Proteins 0.000 description 4
- 102000040430 polynucleotide Human genes 0.000 description 4
- 239000002157 polynucleotide Substances 0.000 description 4
- 239000000243 solution Substances 0.000 description 4
- 239000013603 viral vector Substances 0.000 description 4
- 230000004393 visual impairment Effects 0.000 description 4
- 241000702421 Dependoparvovirus Species 0.000 description 3
- PEDCQBHIVMGVHV-UHFFFAOYSA-N Glycerine Chemical compound OCC(O)CO PEDCQBHIVMGVHV-UHFFFAOYSA-N 0.000 description 3
- 102100038300 Metabotropic glutamate receptor 6 Human genes 0.000 description 3
- 206010029350 Neurotoxicity Diseases 0.000 description 3
- 206010034972 Photosensitivity reaction Diseases 0.000 description 3
- 201000007737 Retinal degeneration Diseases 0.000 description 3
- 208000017442 Retinal disease Diseases 0.000 description 3
- 206010038923 Retinopathy Diseases 0.000 description 3
- 206010044221 Toxic encephalopathy Diseases 0.000 description 3
- 238000010171 animal model Methods 0.000 description 3
- 238000013459 approach Methods 0.000 description 3
- 150000001768 cations Chemical class 0.000 description 3
- 238000001514 detection method Methods 0.000 description 3
- 230000005670 electromagnetic radiation Effects 0.000 description 3
- 238000010304 firing Methods 0.000 description 3
- 230000006872 improvement Effects 0.000 description 3
- 230000004298 light response Effects 0.000 description 3
- 230000033001 locomotion Effects 0.000 description 3
- 238000005259 measurement Methods 0.000 description 3
- 108010038450 metabotropic glutamate receptor 6 Proteins 0.000 description 3
- 238000010172 mouse model Methods 0.000 description 3
- 231100000228 neurotoxicity Toxicity 0.000 description 3
- 230000007135 neurotoxicity Effects 0.000 description 3
- 239000002245 particle Substances 0.000 description 3
- 230000008447 perception Effects 0.000 description 3
- 230000036211 photosensitivity Effects 0.000 description 3
- 230000016732 phototransduction Effects 0.000 description 3
- 230000001179 pupillary effect Effects 0.000 description 3
- 230000004296 scotopic vision Effects 0.000 description 3
- 239000000126 substance Substances 0.000 description 3
- 210000001519 tissue Anatomy 0.000 description 3
- NCYCYZXNIZJOKI-IOUUIBBYSA-N 11-cis-retinal Chemical compound O=C/C=C(\C)/C=C\C=C(/C)\C=C\C1=C(C)CCCC1(C)C NCYCYZXNIZJOKI-IOUUIBBYSA-N 0.000 description 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 description 2
- 108700037328 Chlamydomonas reinhardtii chlamyopsin Proteins 0.000 description 2
- 108020004705 Codon Proteins 0.000 description 2
- 206010010356 Congenital anomaly Diseases 0.000 description 2
- 108010050754 Halorhodopsins Proteins 0.000 description 2
- 102000004310 Ion Channels Human genes 0.000 description 2
- 108090000862 Ion Channels Proteins 0.000 description 2
- 208000010415 Low Vision Diseases 0.000 description 2
- YJPIGAIKUZMOQA-UHFFFAOYSA-N Melatonin Natural products COC1=CC=C2N(C(C)=O)C=C(CCN)C2=C1 YJPIGAIKUZMOQA-UHFFFAOYSA-N 0.000 description 2
- 108091028043 Nucleic acid sequence Proteins 0.000 description 2
- 101710131038 Opsin-2 Proteins 0.000 description 2
- 101710097927 Retinal-binding protein Proteins 0.000 description 2
- NCYCYZXNIZJOKI-OVSJKPMPSA-N Retinaldehyde Chemical compound O=C\C=C(/C)\C=C\C=C(/C)\C=C\C1=C(C)CCCC1(C)C NCYCYZXNIZJOKI-OVSJKPMPSA-N 0.000 description 2
- 108090000820 Rhodopsin Proteins 0.000 description 2
- UIIMBOGNXHQVGW-UHFFFAOYSA-M Sodium bicarbonate Chemical compound [Na+].OC([O-])=O UIIMBOGNXHQVGW-UHFFFAOYSA-M 0.000 description 2
- 230000009471 action Effects 0.000 description 2
- 230000036982 action potential Effects 0.000 description 2
- 210000003050 axon Anatomy 0.000 description 2
- 230000003542 behavioural effect Effects 0.000 description 2
- 230000008901 benefit Effects 0.000 description 2
- 230000005540 biological transmission Effects 0.000 description 2
- 210000003169 central nervous system Anatomy 0.000 description 2
- 230000002060 circadian Effects 0.000 description 2
- 230000001054 cortical effect Effects 0.000 description 2
- 230000006378 damage Effects 0.000 description 2
- 230000034994 death Effects 0.000 description 2
- 230000007547 defect Effects 0.000 description 2
- 230000003292 diminished effect Effects 0.000 description 2
- 238000002474 experimental method Methods 0.000 description 2
- 238000009472 formulation Methods 0.000 description 2
- 108020001507 fusion proteins Proteins 0.000 description 2
- 102000037865 fusion proteins Human genes 0.000 description 2
- 230000036541 health Effects 0.000 description 2
- 230000004305 hyperopia Effects 0.000 description 2
- 201000006318 hyperopia Diseases 0.000 description 2
- 230000002102 hyperpolarization Effects 0.000 description 2
- 230000010365 information processing Effects 0.000 description 2
- 230000002401 inhibitory effect Effects 0.000 description 2
- 230000004446 light reflex Effects 0.000 description 2
- 230000004303 low vision Effects 0.000 description 2
- 230000007246 mechanism Effects 0.000 description 2
- 229960003987 melatonin Drugs 0.000 description 2
- DRLFMBDRBRZALE-UHFFFAOYSA-N melatonin Chemical compound COC1=CC=C2NC=C(CCNC(C)=O)C2=C1 DRLFMBDRBRZALE-UHFFFAOYSA-N 0.000 description 2
- 230000004379 myopia Effects 0.000 description 2
- 208000001491 myopia Diseases 0.000 description 2
- 210000000653 nervous system Anatomy 0.000 description 2
- 210000001328 optic nerve Anatomy 0.000 description 2
- 230000036961 partial effect Effects 0.000 description 2
- 239000008194 pharmaceutical composition Substances 0.000 description 2
- 239000013612 plasmid Substances 0.000 description 2
- 238000002360 preparation method Methods 0.000 description 2
- 230000005855 radiation Effects 0.000 description 2
- 238000011084 recovery Methods 0.000 description 2
- 230000025474 response to light stimulus Effects 0.000 description 2
- 102000024458 retinal binding proteins Human genes 0.000 description 2
- 230000004258 retinal degeneration Effects 0.000 description 2
- 210000003786 sclera Anatomy 0.000 description 2
- 230000001953 sensory effect Effects 0.000 description 2
- 230000008685 targeting Effects 0.000 description 2
- 230000001225 therapeutic effect Effects 0.000 description 2
- 238000002560 therapeutic procedure Methods 0.000 description 2
- 238000010361 transduction Methods 0.000 description 2
- 230000026683 transduction Effects 0.000 description 2
- 241000701161 unidentified adenovirus Species 0.000 description 2
- 230000003612 virological effect Effects 0.000 description 2
- 230000004304 visual acuity Effects 0.000 description 2
- 230000001755 vocal effect Effects 0.000 description 2
- XLYOFNOQVPJJNP-UHFFFAOYSA-N water Substances O XLYOFNOQVPJJNP-UHFFFAOYSA-N 0.000 description 2
- 229910052724 xenon Inorganic materials 0.000 description 2
- FHNFHKCVQCLJFQ-UHFFFAOYSA-N xenon atom Chemical compound [Xe] FHNFHKCVQCLJFQ-UHFFFAOYSA-N 0.000 description 2
- JKMHFZQWWAIEOD-UHFFFAOYSA-N 2-[4-(2-hydroxyethyl)piperazin-1-yl]ethanesulfonic acid Chemical compound OCC[NH+]1CCN(CCS([O-])(=O)=O)CC1 JKMHFZQWWAIEOD-UHFFFAOYSA-N 0.000 description 1
- GOJUJUVQIVIZAV-UHFFFAOYSA-N 2-amino-4,6-dichloropyrimidine-5-carbaldehyde Chemical group NC1=NC(Cl)=C(C=O)C(Cl)=N1 GOJUJUVQIVIZAV-UHFFFAOYSA-N 0.000 description 1
- ZOOGRGPOEVQQDX-UUOKFMHZSA-N 3',5'-cyclic GMP Chemical compound C([C@H]1O2)OP(O)(=O)O[C@H]1[C@@H](O)[C@@H]2N1C(N=C(NC2=O)N)=C2N=C1 ZOOGRGPOEVQQDX-UUOKFMHZSA-N 0.000 description 1
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 description 1
- 206010001258 Adenoviral infections Diseases 0.000 description 1
- 206010005176 Blindness congenital Diseases 0.000 description 1
- OYPRJOBELJOOCE-UHFFFAOYSA-N Calcium Chemical compound [Ca] OYPRJOBELJOOCE-UHFFFAOYSA-N 0.000 description 1
- 208000002177 Cataract Diseases 0.000 description 1
- 108091005462 Cation channels Proteins 0.000 description 1
- 108010035848 Channelrhodopsins Proteins 0.000 description 1
- VEXZGXHMUGYJMC-UHFFFAOYSA-M Chloride anion Chemical compound [Cl-] VEXZGXHMUGYJMC-UHFFFAOYSA-M 0.000 description 1
- 241000195628 Chlorophyta Species 0.000 description 1
- 102000010970 Connexin Human genes 0.000 description 1
- 108050001175 Connexin Proteins 0.000 description 1
- 206010012689 Diabetic retinopathy Diseases 0.000 description 1
- 101100351286 Dictyostelium discoideum pdeE gene Proteins 0.000 description 1
- 206010052804 Drug tolerance Diseases 0.000 description 1
- 208000020564 Eye injury Diseases 0.000 description 1
- 241000282326 Felis catus Species 0.000 description 1
- 208000010412 Glaucoma Diseases 0.000 description 1
- WQZGKKKJIJFFOK-GASJEMHNSA-N Glucose Natural products OC[C@H]1OC(O)[C@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-GASJEMHNSA-N 0.000 description 1
- 239000007995 HEPES buffer Substances 0.000 description 1
- 208000003923 Hereditary Corneal Dystrophies Diseases 0.000 description 1
- 241000282412 Homo Species 0.000 description 1
- 206010020675 Hypermetropia Diseases 0.000 description 1
- 208000026350 Inborn Genetic disease Diseases 0.000 description 1
- 201000002287 Keratoconus 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
- 241000713666 Lentivirus Species 0.000 description 1
- 241000124008 Mammalia Species 0.000 description 1
- 102100025912 Melanopsin Human genes 0.000 description 1
- 101100135809 Mus musculus Pcp2 gene Proteins 0.000 description 1
- 108010021466 Mutant Proteins Proteins 0.000 description 1
- 102000008300 Mutant Proteins Human genes 0.000 description 1
- 239000000020 Nitrocellulose Substances 0.000 description 1
- 102000004861 Phosphoric Diester Hydrolases Human genes 0.000 description 1
- 108090001050 Phosphoric Diester Hydrolases Proteins 0.000 description 1
- 206010038848 Retinal detachment Diseases 0.000 description 1
- 208000032430 Retinal dystrophy Diseases 0.000 description 1
- 239000002262 Schiff base Substances 0.000 description 1
- 150000004753 Schiff bases Chemical class 0.000 description 1
- 206010064930 age-related macular degeneration Diseases 0.000 description 1
- 230000003466 anti-cipated effect Effects 0.000 description 1
- 238000003556 assay Methods 0.000 description 1
- 230000006399 behavior Effects 0.000 description 1
- 238000009227 behaviour therapy Methods 0.000 description 1
- 230000033228 biological regulation Effects 0.000 description 1
- 230000015572 biosynthetic process Effects 0.000 description 1
- 208000002352 blister Diseases 0.000 description 1
- 239000000872 buffer Substances 0.000 description 1
- 239000007975 buffered saline Substances 0.000 description 1
- 229910052791 calcium Inorganic materials 0.000 description 1
- 239000011575 calcium Substances 0.000 description 1
- 230000010221 calcium permeability Effects 0.000 description 1
- 230000003915 cell function Effects 0.000 description 1
- 210000000170 cell membrane Anatomy 0.000 description 1
- 230000001413 cellular effect Effects 0.000 description 1
- 238000006243 chemical reaction Methods 0.000 description 1
- 230000027288 circadian rhythm Effects 0.000 description 1
- 230000004456 color vision Effects 0.000 description 1
- 201000008615 cone dystrophy Diseases 0.000 description 1
- 208000006623 congenital stationary night blindness Diseases 0.000 description 1
- 210000004087 cornea Anatomy 0.000 description 1
- 206010011005 corneal dystrophy Diseases 0.000 description 1
- 230000002596 correlated effect Effects 0.000 description 1
- 230000000875 corresponding effect Effects 0.000 description 1
- 210000000695 crystalline len Anatomy 0.000 description 1
- ZOOGRGPOEVQQDX-UHFFFAOYSA-N cyclic GMP Natural products O1C2COP(O)(=O)OC2C(O)C1N1C=NC2=C1NC(N)=NC2=O ZOOGRGPOEVQQDX-UHFFFAOYSA-N 0.000 description 1
- 230000006735 deficit Effects 0.000 description 1
- 230000003412 degenerative effect Effects 0.000 description 1
- 230000001419 dependent effect Effects 0.000 description 1
- 230000002999 depolarising effect Effects 0.000 description 1
- 230000009547 development abnormality Effects 0.000 description 1
- 230000004069 differentiation Effects 0.000 description 1
- 239000003085 diluting agent Substances 0.000 description 1
- 239000003814 drug Substances 0.000 description 1
- 230000009977 dual effect Effects 0.000 description 1
- 230000002708 enhancing effect Effects 0.000 description 1
- 230000007613 environmental effect Effects 0.000 description 1
- 238000011124 ex vivo culture Methods 0.000 description 1
- 210000001723 extracellular space Anatomy 0.000 description 1
- 230000004424 eye movement Effects 0.000 description 1
- 210000000744 eyelid Anatomy 0.000 description 1
- 230000003371 gabaergic effect Effects 0.000 description 1
- 210000003976 gap junction Anatomy 0.000 description 1
- 238000012239 gene modification Methods 0.000 description 1
- 208000016361 genetic disease Diseases 0.000 description 1
- 230000005017 genetic modification Effects 0.000 description 1
- 235000013617 genetically modified food Nutrition 0.000 description 1
- 239000008103 glucose Substances 0.000 description 1
- 230000000575 glycinergic effect Effects 0.000 description 1
- 230000026781 habituation Effects 0.000 description 1
- 210000003128 head Anatomy 0.000 description 1
- 229940088597 hormone Drugs 0.000 description 1
- 239000005556 hormone Substances 0.000 description 1
- 238000012744 immunostaining Methods 0.000 description 1
- 238000011065 in-situ storage Methods 0.000 description 1
- 230000001939 inductive effect Effects 0.000 description 1
- 210000001153 interneuron Anatomy 0.000 description 1
- 230000031146 intracellular signal transduction Effects 0.000 description 1
- 239000007928 intraperitoneal injection Substances 0.000 description 1
- 229960003299 ketamine Drugs 0.000 description 1
- 230000013016 learning Effects 0.000 description 1
- 230000000670 limiting effect Effects 0.000 description 1
- 208000018769 loss of vision Diseases 0.000 description 1
- 231100000864 loss of vision Toxicity 0.000 description 1
- 230000007257 malfunction Effects 0.000 description 1
- 239000003550 marker Substances 0.000 description 1
- 108010005417 melanopsin Proteins 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
- 238000012986 modification Methods 0.000 description 1
- 235000019799 monosodium phosphate Nutrition 0.000 description 1
- 230000008555 neuronal activation Effects 0.000 description 1
- 230000003472 neutralizing effect Effects 0.000 description 1
- 229920001220 nitrocellulos Polymers 0.000 description 1
- 210000002597 off-bipolar cell Anatomy 0.000 description 1
- 210000001743 on-bipolar cell Anatomy 0.000 description 1
- 210000003977 optic chiasm Anatomy 0.000 description 1
- 210000005112 optic tract Anatomy 0.000 description 1
- 230000004421 optic tracts Effects 0.000 description 1
- 230000003287 optical effect Effects 0.000 description 1
- 230000004466 optokinetic reflex Effects 0.000 description 1
- 101150037969 pde-6 gene Proteins 0.000 description 1
- 230000000737 periodic effect Effects 0.000 description 1
- 230000002085 persistent effect Effects 0.000 description 1
- 239000000546 pharmaceutical excipient Substances 0.000 description 1
- 230000004310 photopic vision Effects 0.000 description 1
- 239000011148 porous material Substances 0.000 description 1
- 238000004321 preservation Methods 0.000 description 1
- 238000012545 processing Methods 0.000 description 1
- 230000000069 prophylactic effect Effects 0.000 description 1
- 210000001747 pupil Anatomy 0.000 description 1
- 239000013608 rAAV vector Substances 0.000 description 1
- 230000009467 reduction Effects 0.000 description 1
- 230000011514 reflex Effects 0.000 description 1
- 238000011160 research Methods 0.000 description 1
- 210000003569 retinal bipolar cell Anatomy 0.000 description 1
- 230000004264 retinal detachment Effects 0.000 description 1
- 230000004243 retinal function Effects 0.000 description 1
- 210000003583 retinal pigment epithelium Anatomy 0.000 description 1
- 210000004761 scalp Anatomy 0.000 description 1
- 230000035945 sensitivity Effects 0.000 description 1
- 230000019491 signal transduction Effects 0.000 description 1
- 229910000030 sodium bicarbonate Inorganic materials 0.000 description 1
- 235000017557 sodium bicarbonate Nutrition 0.000 description 1
- AJPJDKMHJJGVTQ-UHFFFAOYSA-M sodium dihydrogen phosphate Chemical compound [Na+].OP(O)([O-])=O AJPJDKMHJJGVTQ-UHFFFAOYSA-M 0.000 description 1
- 229910000162 sodium phosphate Inorganic materials 0.000 description 1
- 238000006467 substitution reaction Methods 0.000 description 1
- 239000000758 substrate Substances 0.000 description 1
- 239000000725 suspension Substances 0.000 description 1
- 208000024891 symptom Diseases 0.000 description 1
- 230000000946 synaptic effect Effects 0.000 description 1
- 230000009466 transformation Effects 0.000 description 1
- 241001430294 unidentified retrovirus Species 0.000 description 1
- 239000003981 vehicle Substances 0.000 description 1
- 210000002845 virion Anatomy 0.000 description 1
- 238000002645 vision therapy Methods 0.000 description 1
- 230000003945 visual behavior Effects 0.000 description 1
- 230000004382 visual function Effects 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
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/405—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from algae
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives 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
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Organic Chemistry (AREA)
- Medicinal Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Molecular Biology (AREA)
- Genetics & Genomics (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Biotechnology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Ophthalmology & Optometry (AREA)
- Immunology (AREA)
- Epidemiology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261606663P | 2012-03-05 | 2012-03-05 | |
| PCT/US2013/029171 WO2013134295A1 (en) | 2012-03-05 | 2013-03-05 | Identification of channelrhodopsin-2 (chop2) mutations and methods of use |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK2822964T3 true DK2822964T3 (en) | 2018-12-10 |
Family
ID=47892055
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK13710253.9T DK2822964T3 (en) | 2012-03-05 | 2013-03-05 | IDENTIFICATION OF CHANNEL RHODOPSIN-2- (CHOP2) MUTATIONS AND METHODS OF USE |
Country Status (16)
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8470790B2 (en) | 2006-05-04 | 2013-06-25 | Wayne State University | Restoration of visual responses by in vivo delivery of rhodopsin nucleic acids |
| WO2011140279A1 (en) | 2010-05-04 | 2011-11-10 | Wayne State University | Aav-mediated subcellular targeting of heterologous rhodopsins in retinal ganglion cells |
| EA201491643A1 (ru) | 2012-03-05 | 2015-02-27 | Уэйн Стейт Юниверсити | Идентификация мутаций канал-опсина-2 (chop2) и способы применения |
| AU2014243925B2 (en) | 2013-03-14 | 2018-12-06 | Wayne State University | Method of enhancing delivery of therapeutic compounds to the eye |
| JP6469669B2 (ja) | 2013-07-08 | 2019-02-13 | ビーエーエスエフ ソシエタス・ヨーロピアＢａｓｆ Ｓｅ | オキシムエステル光開始剤 |
| US10307492B2 (en) | 2014-03-11 | 2019-06-04 | Wayne State University | Modified MGLUR6 promoter and methods of use |
| EP3452067A1 (en) | 2016-05-03 | 2019-03-13 | Wayne State University | Method of enhancing viral-mediated gene delivery in the eye using proteosome inhibitors |
| EP3464341A1 (en) * | 2016-06-03 | 2019-04-10 | Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. | Mutant light-inducible ion channel of channelrhodopsin |
| WO2018044912A1 (en) * | 2016-08-29 | 2018-03-08 | Wayne State University | Identification of mutations in channelopsin variants having improved light sensitivity and methods of use thereof |
| EP3535282A4 (en) * | 2016-11-06 | 2020-04-08 | Nanoscope Technologies LLC | OPTOGENETIC MODULATION BY OPSINS WITH MULTIPLE CHARACTERISTICS FOR RESTORATION OF VISION AND OTHER ASSOCIATED APPLICATIONS |
| US20200115419A1 (en) * | 2017-04-12 | 2020-04-16 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | New optogenetic tool |
| PL3720428T3 (pl) * | 2017-12-04 | 2023-05-08 | Laboratorios Salvat, S.A. | Kompozycja oftalmiczna do podawania miejscowego zawierająca kwas dobezylowy do leczenia chorób tylnego odcinka oka |
| JP6567153B1 (ja) * | 2018-09-28 | 2019-08-28 | 学校法人東北工業大学 | 対象化合物の特性の予測を行うための方法、コンピュータシステム、プログラム |
| WO2024226667A2 (en) * | 2023-04-24 | 2024-10-31 | Nanoscope Therapeutics Inc. | Optogenetic modulation by multi-characteristic opsins for vision restoration and other applications thereof |
Family Cites Families (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4554101A (en) | 1981-01-09 | 1985-11-19 | New York Blood Center, Inc. | Identification and preparation of epitopes on antigens and allergens on the basis of hydrophilicity |
| US4498919A (en) | 1983-10-31 | 1985-02-12 | Corning Glass Works | Method for making colored photochromic prescription ophthalmic lenses |
| US6610287B1 (en) | 1990-04-16 | 2003-08-26 | The General Hospital Corporation | Transfer and expression of gene sequences into nervous system cells using herpes simplex virus mutants with deletions in genes for viral replication |
| US5827702A (en) | 1994-10-31 | 1998-10-27 | Genentech, Inc. | Ocular gene therapy |
| EP0977880A2 (en) | 1997-04-21 | 2000-02-09 | University Of Florida | Materials and methods for treatment of retinal diseases |
| CA2346262A1 (en) | 1998-09-17 | 2000-03-23 | University Of Florida | Methods for treatment of degenerative retinal diseases |
| JP2004514407A (ja) | 2000-04-28 | 2004-05-20 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 異種キャプシド中にシュードタイピングされたａａｖ５キャプシドおよびａａｖ５ベクターを含む組換えａａｖベクター |
| CA2442670A1 (en) | 2001-04-13 | 2002-10-24 | The Trustees Of The University Of Pennsylvania | Method of treating or retarding the development of blindness |
| AU2002323210A1 (en) | 2001-08-16 | 2003-03-03 | Sloan Kettering Institute For Cancer Research | Bio-synthetic photostimulators and methods of use |
| DE10216005A1 (de) | 2002-04-11 | 2003-10-30 | Max Planck Gesellschaft | Verwendung von biologischen Photorezeptoren als direkt lichtgesteuerte Ionenkanäle |
| AU2003249318A1 (en) | 2002-07-18 | 2004-02-09 | The General Hospital Corp. | Method for augmenting vision in persons suffering from photoreceptor cell degeneration |
| US7186699B2 (en) | 2003-06-03 | 2007-03-06 | Cell Genesys, Inc. | Method for treating cancer by vector-mediated delivery of one or more anti-angiogenic or pro-apoptotic genes |
| WO2005044096A2 (en) | 2003-11-05 | 2005-05-19 | Neurovision, Inc. | Method for improving visual perception |
| JP2009502140A (ja) | 2005-07-22 | 2009-01-29 | ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー | 光活性化陽イオンチャネルおよびその使用 |
| US8470790B2 (en) | 2006-05-04 | 2013-06-25 | Wayne State University | Restoration of visual responses by in vivo delivery of rhodopsin nucleic acids |
| WO2011140279A1 (en) * | 2010-05-04 | 2011-11-10 | Wayne State University | Aav-mediated subcellular targeting of heterologous rhodopsins in retinal ganglion cells |
| HRP20150846T1 (hr) * | 2010-09-08 | 2015-09-11 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | Mutirani kalanski rodopsin 2 |
| EA201491643A1 (ru) * | 2012-03-05 | 2015-02-27 | Уэйн Стейт Юниверсити | Идентификация мутаций канал-опсина-2 (chop2) и способы применения |
| KR101639859B1 (ko) * | 2014-03-06 | 2016-07-14 | 포항공과대학교 산학협력단 | 광 자극에 의한 미토콘드리아 활성의 시공간적 가역 조절 방법 |
-
2013
- 2013-03-05 EA EA201491643A patent/EA201491643A1/ru unknown
- 2013-03-05 MX MX2014010664A patent/MX360085B/es active IP Right Grant
- 2013-03-05 ES ES13710253T patent/ES2703324T3/es active Active
- 2013-03-05 PL PL13710253T patent/PL2822964T3/pl unknown
- 2013-03-05 NZ NZ629843A patent/NZ629843A/en unknown
- 2013-03-05 KR KR1020147027985A patent/KR102137717B1/ko active Active
- 2013-03-05 EP EP13710253.9A patent/EP2822964B1/en active Active
- 2013-03-05 WO PCT/US2013/029171 patent/WO2013134295A1/en not_active Ceased
- 2013-03-05 JP JP2014561051A patent/JP6395611B2/ja active Active
- 2013-03-05 CN CN201380022635.5A patent/CN104334575A/zh active Pending
- 2013-03-05 HU HUE13710253A patent/HUE040487T2/hu unknown
- 2013-03-05 CN CN202010025519.0A patent/CN111378020A/zh active Pending
- 2013-03-05 AU AU2013230112A patent/AU2013230112A1/en not_active Abandoned
- 2013-03-05 PT PT13710253T patent/PT2822964T/pt unknown
- 2013-03-05 CA CA2866405A patent/CA2866405A1/en active Pending
- 2013-03-05 DK DK13710253.9T patent/DK2822964T3/en active
- 2013-03-05 US US14/383,211 patent/US10947281B2/en active Active
-
2017
- 2017-10-06 AU AU2017239601A patent/AU2017239601B2/en active Active
-
2018
- 2018-04-23 JP JP2018082403A patent/JP2018134098A/ja not_active Withdrawn
-
2019
- 2019-07-01 JP JP2019123061A patent/JP2019193656A/ja active Pending
- 2019-08-14 AU AU2019216638A patent/AU2019216638B2/en active Active
-
2021
- 2021-03-15 US US17/202,064 patent/US20220033449A1/en not_active Abandoned
- 2021-10-07 AU AU2021245195A patent/AU2021245195B2/en active Active
-
2024
- 2024-04-18 US US18/639,381 patent/US20250066431A1/en active Pending
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US20250066431A1 (en) | Identification of channelrhodopsin-2 (chr2) mutations and methods of use | |
| US20240165198A1 (en) | Optogenetic visual restoration using chrimson | |
| ES2538468T3 (es) | Vectores para la administración de proteínas sensibles a la luz y métodos para su utilización | |
| JP6594854B2 (ja) | 行動状態の光遺伝学的制御方法 | |
| KR102008538B1 (ko) | 감광성 키메라 gpcr 단백질 | |
| JP2009536219A (ja) | ロドプシン拡散のインビボ送達による視覚応答の回復 | |
| US20230159609A1 (en) | G-protein-gated-k+ channel-mediated enhancements in light sensitivity in rod-cone dystrophy (rcd) | |
| JP6779546B2 (ja) | 視力回復のための多特性オプシンによる光遺伝学的調節及びその別の用途 | |
| EP3892738A1 (en) | G-protein-gated-k+ channel-mediated enhancements in light sensitivity in rod-cone dystrophy (rcd) | |
| HK1205744B (en) | Identification of channelrhodopsin-2 (chop2) mutations and methods of use | |
| EA043449B1 (ru) | ИДЕНТИФИКАЦИЯ МУТАЦИЙ КАНАЛ-ОПСИНА-2 (Chop2) И СПОСОБЫ ПРИМЕНЕНИЯ | |
| US20230338581A1 (en) | G-protein-gated-k+ channel-mediated enhancements in light sensitivity in rod-cone dystrophy (rcd) | |
| NZ727041B2 (en) | Identification of channelrhodopsin-2 (chop2) mutations and methods of use |