Safe and effective superoxide dismutase 1 silencing using artificial microRNA in macaques-Reference-Cited by-同舟云学术

Safe and effective superoxide dismutase 1 silencing using artificial microRNA in macaques

Author:

Borel Florie¹²^ORCID,Gernoux Gwladys¹^ORCID,Sun Huaming¹^ORCID,Stock Rachel¹^ORCID,Blackwood Meghan¹^ORCID,Brown Robert H.³,Mueller Christian¹⁴

Affiliation:

1. Horae Gene Therapy Center, University of Massachusetts Medical School, 368 Plantation Street, Worcester, MA 01605, USA.

2. Shire, 125 Binney Street, Cambridge, MA 02142, USA.

3. Department of Neurology, University of Massachusetts Medical School, 368 Plantation Street, Worcester, MA 01605, USA.

4. Department of Pediatrics, University of Massachusetts Medical School, 368 Plantation Street, Worcester, MA 01605, USA.

Abstract

Intrathecal delivery of artificial microRNA reduced SOD1 expression in motor neurons in macaques.

Funder

Alpha-1 Foundation

Innovative Structural Materials Association

NINDS

ALS Alliance Therapy

Rizzuto fund

Angel Fund for ALS Research

Pierre L. de Bourgknecht ALS Research Foundation

Project ALS

P2ALS

ALS-FindingACure

ALSOne

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

Reference41 articles.

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