AR097920A1 - COMPOSITIONS AND METHODS TO INHIBIT THE EXPRESSION OF GENE SINTASA 1 OF 5-AMINOLEVULIN ACID (ALAS1) - Google Patents

COMPOSITIONS AND METHODS TO INHIBIT THE EXPRESSION OF GENE SINTASA 1 OF 5-AMINOLEVULIN ACID (ALAS1)

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Publication number
AR097920A1
AR097920A1 ARP140103699A ARP140103699A AR097920A1 AR 097920 A1 AR097920 A1 AR 097920A1 AR P140103699 A ARP140103699 A AR P140103699A AR P140103699 A ARP140103699 A AR P140103699A AR 097920 A1 AR097920 A1 AR 097920A1
Authority
AR
Argentina
Prior art keywords
alas1
expression
compositions
dsrna
inhibit
Prior art date
Application number
ARP140103699A
Other languages
Spanish (es)
Original Assignee
Icahn School Med Mount Sinai
Alnylam Pharmaceuticals Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Icahn School Med Mount Sinai, Alnylam Pharmaceuticals Inc filed Critical Icahn School Med Mount Sinai
Publication of AR097920A1 publication Critical patent/AR097920A1/en

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Abstract

Se refiere a composiciones de ácido ribonucleico bicatenario (dsARN) opcionalmente conjugado a un ligando tal como N-acetilgalactosamina. Los dsARN están dirigidos al gen ALAS1. Métodos de uso de tales composiciones de dsARN para alterar (por ejemplo, inhibir) la expresión de ALAS1, tal como tratamientos de sujetos con porfiria. Reivindicación 1: Un ácido ribonucleico bicatenario (dsARN) para inhibir la expresión de ALAS1, en donde dicho dsARN comprende una cadena con sentido y una cadena antisentido, donde la cadena antisentido comprende una región de complementariedad con un transcripto de ARN de ALAS1 (por ejemplo, SEQ ID N º1), cuya cadena antisentido comprende al menos 20 nucleótidos contiguos de la secuencia antisentido de UAAGAUGAGACACUCUUUCUGGU (SEQ ID Nº 4153) o UAAGAUGAGACACUCTUUCUGGU (SEQ ID Nº 4154).It refers to double stranded ribonucleic acid (dsRNA) compositions optionally conjugated to a ligand such as N-acetylgalactosamine. The dsRNAs are directed to the ALAS1 gene. Methods of using such dsRNA compositions to alter (for example, inhibit) the expression of ALAS1, such as treatments of subjects with porphyria. Claim 1: A double stranded ribonucleic acid (dsRNA) for inhibiting the expression of ALAS1, wherein said dsRNA comprises a sense chain and an antisense chain, wherein the antisense chain comprises a region of complementarity with an ALAS1 RNA transcript (for example , SEQ ID No. 1), whose antisense chain comprises at least 20 contiguous nucleotides of the antisense sequence of UAAGAUGAGACACUCUUUCUGGU (SEQ ID No. 4153) or UAAGAUGAGACACUCTUUCUGGU (SEQ ID No. 4154).

ARP140103699A 2013-10-04 2014-10-03 COMPOSITIONS AND METHODS TO INHIBIT THE EXPRESSION OF GENE SINTASA 1 OF 5-AMINOLEVULIN ACID (ALAS1) AR097920A1 (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201361887288P 2013-10-04 2013-10-04

Publications (1)

Publication Number Publication Date
AR097920A1 true AR097920A1 (en) 2016-04-20

Family

ID=58698725

Family Applications (2)

Application Number Title Priority Date Filing Date
ARP140103699A AR097920A1 (en) 2013-10-04 2014-10-03 COMPOSITIONS AND METHODS TO INHIBIT THE EXPRESSION OF GENE SINTASA 1 OF 5-AMINOLEVULIN ACID (ALAS1)
ARP200101035A AR118658A2 (en) 2013-10-04 2020-04-13 COMPOSITIONS AND METHODS TO INHIBIT THE EXPRESSION OF THE ALAS1 GENE

Family Applications After (1)

Application Number Title Priority Date Filing Date
ARP200101035A AR118658A2 (en) 2013-10-04 2020-04-13 COMPOSITIONS AND METHODS TO INHIBIT THE EXPRESSION OF THE ALAS1 GENE

Country Status (2)

Country Link
AR (2) AR097920A1 (en)
MA (1) MA39000B1 (en)

Also Published As

Publication number Publication date
AR118658A2 (en) 2021-10-20
MA39000B1 (en) 2018-12-31
MA39000A1 (en) 2018-05-31

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