DK2776455T3 - Kanalrhodopsiner til optisk kontrol af celler - Google Patents
Kanalrhodopsiner til optisk kontrol af celler Download PDFInfo
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Claims (15)
1. Isoleret, lysaktiveret ionkanal-polypeptid omfattende en aminosyresekvens af et vildtype eller modificeret lysaktiveret Chlamydomonas noctigama-kanal-polypeptid, hvor aminosyresekvensen af det lysaktiverede ionkanal-polypeptid omfatter SEQ ID NO:2 eller aminosyresekvensen af det lysaktiverede ionkanal-polypeptid omfatter en modificeret Chlamydomonas noctigama lysaktiveret ionkanal-sekvens, der har mindst 70 % identitet til aminosyrer 86-320 af SEQ ID NO: 2 og mindst 95 % identitet til de resterende aminosyrer i sekvensen angivet som SEQ ID NO:2, hvorved en nukleinsyre- eller polypeptid-sekvens kan blive naturligt udtrykt i en celle eller organisme af et medlem af Chlamydomonas-s\ægten, men forudsat at sekvensen ikke er en del af eller inkluderet i en Chlamydomonas-ceWe eller -organisme, den er betragtet at være isoleret fra.
2. Polypeptidet ifølge krav 1, hvor aminosyresekvensen af det lysaktiverede ionkanal-polypeptid omfatter en modificeret Chlamydomonas noctigama lysaktiveret ionkanal-sekvens, der har mindst 75 %, 80 %, 85 %, 90 %, 95 %, eller 99 % identitet til aminosyrer 86-320 af SEQ ID NO: 2 og mindst 96 %, 97 %, 98 %, 99 % eller 100 % identitet til de resterende aminosyrer i sekvensen angivet som SEQ ID NO:2.
3. Polypeptidet ifølge krav 1, hvor aminosyresekvensen af det lysaktiverede ionkanal-polypeptid omfatter SEQ ID NO:5.
4. Polypeptidet ifølge krav 1, hvor aminosyresekvensen af det lysaktiverede ionkanal-polypeptid omfatter en modificeret Chlamydomonas noctigama lysaktiveret ionkanal-sekvens, der har mindst 70 %, 75 %, 80 %, 85 %, 90 %, 95 %, eller 99 % identitet til aminosyrer 86-320 af SEQ ID NO: 5 og 95 %, 96 %, 97 %, 98 %, 99 % eller 100 % identitet til de resterende aminosyrer i sekvensen angivet som SEQ ID NO:5.
5. Nukleinsyre, der koder det isolerede, lysaktiverede ionkanal-polypeptid som defineret i et hvilket som helst af kravene 1-4.
6. Nukleinsyren ifølge krav 5, hvor nukleinsyresekvensen er som angivet i SEQ ID NO:3.
7. Vektor omfattende nukleinsyren ifølge krav 5 eller 6.
8. Vektoren ifølge krav 7, hvor vektoren er en ekspressionsvektor.
9. Det lysaktiverede ionkanal-polypeptid som defineret i et hvilket som helst af kravene 1-4 til anvendelse i en fremgangsmåde til medicinsk behandling af et individ.
10. Polypeptidet til anvendelse i en fremgangsmåde ifølge krav 9, hvor lidelsen er valgt fra alkalose og lidelser af synssystemet.
11. Lysaktiveret ionkanal-polypeptid ifølge et hvilket som helst af kravene 1-4, en nukleinsyre ifølge kravene 5-6, eller en vektor ifølge kravene 7-8 til anvendelse i en fremgangsmåde til behandling afen lidelse i et individ, hvilken fremgangsmåde omfatter: a) at administrere til et individ med behov for sådan behandling, en terapeutisk effektiv mængde af det lysaktiverede ionkanal-polypeptid, nukleinsyren eller vektoren, for at behandle lidelsen, hvorved administrationen resulterer i udtryk af den lysaktiverede ionkanal i en celle i individet, og b) at bringe cellen af individet i kontakt med lys og at aktivere den lysaktiverede ionkanal i cellen underforhold tilstrækkelige til at ændre ion-ledeevne af en cellemembran, hvor ændring af ledeevnen af cellemembranen behandler lidelsen.
12. Polypeptidet, nukleinsyren eller vektoren til anvendelse i en fremgangsmåde ifølge krav 11, hvor at ændre ion-ledeevnen af membranen depolariserer cellen.
13. Polypeptidet, nukleinsyre, eller vektor til anvendelse i en fremgangsmåde ifølge krav 11, hvor lidelsen er valgt fra skade, hjerneskade og degenerative neurologiske tilstande.
14. Polypeptidet, nukleinsyren eller vektoren til anvendelse i en fremgangsmåde ifølge krav 13, hvor lidelsen er valgt fra Parkinsons sygdom, Alzheimers sygdom, slagtilfælde, synstab og høretab.
15. Fremgangsmåde til at ændre ion-ledeevne af en membran, hvilken fremgangsmåde omfatter: a) at udtrykke i en værtscellemembran et lysaktiveret ionkanal-polypeptid som defineret i et hvilket som helst af kravene 1-4, og b) at bringe det lysaktiverede ionkanal-polypeptid i kontakt med et lys, der aktiverer den lysaktiverede ionkanal og ændrer ion-ledeevnen af membranen, hvor fremgangsmåden udføres in vitro.
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DK3214094T3 (da) * | 2011-11-12 | 2020-07-13 | Massachusetts Inst Technology | Kanalrhodopsiner til optisk kontrol af celler |
WO2015167639A1 (en) * | 2014-02-07 | 2015-11-05 | Massachusetts Institute Of Technology | Blue light-activated ion channel molecules and uses thereof |
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US10882892B2 (en) | 2014-08-05 | 2021-01-05 | Massachusetts Institute Of Technology | Channelrhodopsin variants and uses thereof |
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