JPWO2019138250A5 - - Google Patents

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Publication number

JPWO2019138250A5

JPWO2019138250A5 JP2020538584A JP2020538584A JPWO2019138250A5 JP WO2019138250 A5 JPWO2019138250 A5 JP WO2019138250A5 JP 2020538584 A JP2020538584 A JP 2020538584A JP 2020538584 A JP2020538584 A JP 2020538584A JP WO2019138250 A5 JPWO2019138250 A5 JP WO2019138250A5

Authority

JP

Japan

Prior art keywords

seq

sequence

tcu

vector

nucleotides

Prior art date

2022-01-21

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.)

Granted

Links

• Espacenet
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• 125000003729 nucleotide group Chemical group 0.000 claims description 19
• 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 11
• 108091008695 photoreceptors Proteins 0.000 claims description 11
• 210000000234 capsid Anatomy 0.000 claims description 8
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• 208000017442 Retinal disease Diseases 0.000 claims description 7
• 239000008194 pharmaceutical composition Substances 0.000 claims description 6
• 238000011282 treatment Methods 0.000 claims description 5
• 241000580270 Adeno-associated virus - 4 Species 0.000 claims description 4
• 102100029142 Cyclic nucleotide-gated cation channel alpha-3 Human genes 0.000 claims description 4
• 101000771071 Homo sapiens Cyclic nucleotide-gated cation channel alpha-3 Proteins 0.000 claims description 4
• 239000013603 viral vector Substances 0.000 claims description 4
• 208000006992 Color Vision Defects Diseases 0.000 claims description 2
• 102100039484 Cone cGMP-specific 3',5'-cyclic phosphodiesterase subunit alpha' Human genes 0.000 claims description 2
• 102100029140 Cyclic nucleotide-gated cation channel beta-3 Human genes 0.000 claims description 2
• 241000702421 Dependoparvovirus Species 0.000 claims description 2
• 102100039214 Guanine nucleotide-binding protein G(t) subunit alpha-2 Human genes 0.000 claims description 2
• 101000609790 Homo sapiens Cone cGMP-specific 3',5'-cyclic phosphodiesterase subunit alpha' Proteins 0.000 claims description 2
• 101000771083 Homo sapiens Cyclic nucleotide-gated cation channel beta-3 Proteins 0.000 claims description 2
• 101000888142 Homo sapiens Guanine nucleotide-binding protein G(t) subunit alpha-2 Proteins 0.000 claims description 2
• 101000943985 Homo sapiens Potassium voltage-gated channel subfamily V member 2 Proteins 0.000 claims description 2
• 101000710852 Homo sapiens Retinal cone rhodopsin-sensitive cGMP 3',5'-cyclic phosphodiesterase subunit gamma Proteins 0.000 claims description 2
• 101000740765 Homo sapiens Voltage-dependent calcium channel subunit alpha-2/delta-4 Proteins 0.000 claims description 2
• 102100033492 Potassium voltage-gated channel subfamily V member 2 Human genes 0.000 claims description 2
• 102100033844 Retinal cone rhodopsin-sensitive cGMP 3',5'-cyclic phosphodiesterase subunit gamma Human genes 0.000 claims description 2
• 102100037053 Voltage-dependent calcium channel subunit alpha-2/delta-4 Human genes 0.000 claims description 2
• 201000007254 color blindness Diseases 0.000 claims description 2
• 239000003937 drug carrier Substances 0.000 claims description 2
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• 230000022532 regulation of transcription, DNA-dependent Effects 0.000 claims 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 4
• 238000000034 method Methods 0.000 description 4
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• 210000000964 retinal cone photoreceptor cell Anatomy 0.000 description 3
• 238000004519 manufacturing process Methods 0.000 description 1
• 108020004707 nucleic acids Proteins 0.000 description 1
• 150000007523 nucleic acids Chemical class 0.000 description 1
• 102000039446 nucleic acids Human genes 0.000 description 1
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• 230000001105 regulatory effect Effects 0.000 description 1
• 210000001525 retina Anatomy 0.000 description 1
• 230000002103 transcriptional effect Effects 0.000 description 1