[go: up one dir, main page]

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)

Info

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

Links

Landscapes

  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)

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

Similar Documents

Publication Publication Date Title
CO2017007335A2 (en) Suppression of the arni-induced huntingtin gene
MX2018006989A (en) Methods and compositions for treating a serpinc1-associated disorder.
CL2023002870A1 (en) Arni compositions against angiotensinogen (agt) and methods for their use
AR090869A1 (en) SERPINC1 RNAi COMPOSITIONS AND THEIR METHODS OF USE
PE20210921A1 (en) ARNI AGENTS AS INHIBITORS OF SERPINE 1 TRANSCRIPTION AND COMPOSITIONS THAT INCLUDE THEM
MX385048B (en) COMPOSITIONS AND METHODS FOR INHIBITING THE EXPRESSION OF THE ALAS1 GENE.
CR20150493A (en) AMRI COMPOSITIONS AGAINST COMPONENT C5 OF THE COMPLEMENT AND METHODS FOR USE
EA201991369A1 (en) MODIFIED RNA GUIDES
DOP2022000085A (en) COMPOSITIONS AND METHODS TO INHIBIT THE EXPRESSION OF THE ALAS1 GENE
PE20171763A1 (en) COMPOSITIONS AND METHODS TO INHIBIT THE EXPRESSION OF THE HAO1 GENE (HYDROXIACIDE-OXIDASE 1 (GLYCOLATE-OXIDASE))
MX2016016905A (en) Nuclease-mediated dna assembly.
AR100946A1 (en) COMPOSITIONS AND METHODS TO REGULATE GENE EXPRESSION THROUGH RNA INTERFERENCE
MX385871B (en) COMPOSITIONS AND METHODS FOR INHIBITING THE EXPRESSION OF THE LECT2 GENE.
MX381946B (en) MULTIPLE OLIGONUCLEOTIDE MOISTURES ON PEPTIDE SUPPORT.
AR083445A1 (en) siRNA AGAINST FIBROSIS
AR079649A1 (en) COMPOSITIONS AND METHODS TO INHIBIT THE EXPRESSION OF GENES IL-18
AR118658A2 (en) COMPOSITIONS AND METHODS TO INHIBIT THE EXPRESSION OF THE ALAS1 GENE
AR133772A2 (en) COMPOSITIONS AND METHODS TO INHIBIT THE EXPRESSION OF THE ALAS1 GENE
UA114603C2 (en) P0 gene silencing constructs and use
AR088562A1 (en) METHODS AND COMPOSITIONS TO SILENCE GENES USING MICRO ARN ARTIFICIALS
AR090582A1 (en) METHODS AND COMPOSITIONS TO TREAT CANCER USING ARNhc SRC 3 BIFUNCTIONAL
AR132165A2 (en) COMPOSITIONS AND METHODS FOR INHIBITING HAO1 (HYDROXY ACID OXIDASE 1 (GLYCOLATE OXIDASE)) GENE EXPRESSION
AR095559A1 (en) MONOTHERAPY WITH ANTI-CLUSTERINE OLIGONUCLEOTIDES TO TREAT CANCER
AR127137A2 (en) SERPINC1 ARNI COMPOSITIONS AND THEIR METHODS OF USE
AR121850A2 (en) LPA RNA INTERFERENCE AGENT (RNAi), USE OF SAID AGENT AND METHOD FOR INHIBITING LPA EXPRESSION

Legal Events

Date Code Title Description
FG Grant, registration