Rescue of a Mouse Model of Spinal Muscular Atrophy With Respiratory Distress Type 1 by AAV9-IGHMBP2 Is Dose Dependent-Reference-Cited by-同舟云学术

Rescue of a Mouse Model of Spinal Muscular Atrophy With Respiratory Distress Type 1 by AAV9-IGHMBP2 Is Dose Dependent

Published:2016-05 Issue:5 Volume:24 Page:855-866
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Shababi Monir,Feng Zhihua,Villalon Eric,Sibigtroth Christine M,Osman Erkan Y,Miller Madeline R,Williams-Simon Patricka A,Lombardi Abby,Sass Thalia H,Atkinson Arleigh K,Garcia Michael L,Ko Chien-Ping,Lorson Christian L

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference34 articles.

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3. Diaphragmatic spinal muscular atrophy with respiratory distress is heterogeneous, and one form Is linked to chromosome 11q13-q21;Grohmann;Am J Hum Genet,1999

4. Mutations in the gene encoding immunoglobulin mu-binding protein 2 cause spinal muscular atrophy with respiratory distress type 1;Grohmann;Nat Genet,2001

5. The ultrastructure of peripheral nerve, motor end-plate and skeletal muscle in patients suffering from spinal muscular atrophy with respiratory distress type 1 (SMARD1);Diers;Acta Neuropathol,2005

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