AR120712A1 - HUMAN CHROMOSOME 9 OPEN READING FRAME 72 (C9ORF72) RNAi AGENTS, COMPOSITIONS AND METHODS OF USE THEREOF - Google Patents

HUMAN CHROMOSOME 9 OPEN READING FRAME 72 (C9ORF72) RNAi AGENTS, COMPOSITIONS AND METHODS OF USE THEREOF

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Publication number
AR120712A1
AR120712A1 ARP200103433A ARP200103433A AR120712A1 AR 120712 A1 AR120712 A1 AR 120712A1 AR P200103433 A ARP200103433 A AR P200103433A AR P200103433 A ARP200103433 A AR P200103433A AR 120712 A1 AR120712 A1 AR 120712A1
Authority
AR
Argentina
Prior art keywords
c9orf72
methods
compositions
reading frame
open reading
Prior art date
Application number
ARP200103433A
Other languages
Spanish (es)
Inventor
Elane Fishilevich
Stuart Milstein
Kirk Brown
Tracy Zimmermann
James D Mininch
David Frendewey
Eric Chiao
Aarti Sharma-Kanning
Anthony Gagliardi
Gustavo Droguett
Brittany Dubose
Brian Zambrowicz
Original Assignee
Alnylam Pharmaceuticals Inc
Regeneron Pharma
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 Alnylam Pharmaceuticals Inc, Regeneron Pharma filed Critical Alnylam Pharmaceuticals Inc
Publication of AR120712A1 publication Critical patent/AR120712A1/en

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Abstract

Agentes y composiciones de ácido ribonucleico de hebra doble (ARNhdi) dirigidos al gen del marco de lectura abierto 72 del cromosoma 9 humano (C9orf72), así como métodos para inhibir la expresión de un gen C9orf72 y métodos de tratamiento de sujetos que padecen una enfermedad o trastorno asociado a C9orf72, por ejemplo, esclerosis lateral amiotrófica / demencia frontotemporal por C9orf72 o el síndrome tipo Huntington debido a expansiones del C9orf72, usando dichos agentes de ARNhdi y composiciones con los mismos.Double-stranded ribonucleic acid (dsRNA) agents and compositions targeting the human chromosome 9 open reading frame 72 (C9orf72) gene, as well as methods for inhibiting the expression of a C9orf72 gene and methods of treating subjects suffering from a disease or C9orf72-associated disorder, eg, C9orf72 amyotrophic lateral sclerosis/frontotemporal dementia or Huntington-like syndrome due to C9orf72 expansions, using such shRNA agents and compositions therewith.

ARP200103433A 2019-12-13 2020-12-10 HUMAN CHROMOSOME 9 OPEN READING FRAME 72 (C9ORF72) RNAi AGENTS, COMPOSITIONS AND METHODS OF USE THEREOF AR120712A1 (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201962947605P 2019-12-13 2019-12-13

Publications (1)

Publication Number Publication Date
AR120712A1 true AR120712A1 (en) 2022-03-09

Family

ID=80994848

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP200103433A AR120712A1 (en) 2019-12-13 2020-12-10 HUMAN CHROMOSOME 9 OPEN READING FRAME 72 (C9ORF72) RNAi AGENTS, COMPOSITIONS AND METHODS OF USE THEREOF

Country Status (1)

Country Link
AR (1) AR120712A1 (en)

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