A ray of hope for ALS patients

Amyotrophic laterel sclerosis(ALS) is one of the most miserable disease. With the degeneration of the lower motor neuron, the patient with this illness should undergo gradual loss of motor functions while the peception and intellectual functions are preserved. The patient, fully alert and conscious, should go on dying for a few years.

 

In my med student days, the cause as well as the treatment were totally unknown. Only symptomatic treatments have been given to the patients. But the recent development in gene analysis revealed the ADAR2 is the defect in this illness. This paper shows they could convey ADAR2 to the neural tissue with a vector AAV9. 

 

It should be confirmed thaty this vector is not harmful to the body. Once it is confirmed safe, this research result could be a real epoch making. Really amazing.

 

I hope this will give the patients a real hope for cure very soon.

 

I am thrilled and pleased to see the medical science is developing in this way. The human beings should spend time, money and their intelligence in such a field, but not in developing weapons etc.

 

quote;

 

EMBO Mol Med. 2013 Sep 24. doi: 10.1002/emmm.201302935. [Epub ahead of print]

Rescue of amyotrophic lateral sclerosis phenotype in a mouse model by intravenous AAV9-ADAR2 delivery to motor neurons.

Yamashita T, Chai HL, Teramoto S, Tsuji S, Shimazaki K, Muramatsu SI, Kwak S.

Source

CREST, Japan Science and Technology Agency, Graduate School of Medicine, University of Tokyo, Bunkyo-Ku, Tokyo, Japan; Department of Neurology, Graduate School of Medicine, University of Tokyo, Bunkyo-Ku, Tokyo, Japan; Center for Disease Biology and Integrative Medicine, Graduate School of Medicine, The University of Tokyo, Bunkyo-Ku, Tokyo, Japan.

Abstract

Amyotrophic lateral sclerosis (ALS) is the most common adult-onset motor neuron disease, and the lack of effective therapy results in inevitable death within a few years of onset. Failure of GluA2 RNA editing resulting from downregulation of the RNA-editing enzyme adenosine deaminase acting on RNA 2 (ADAR2) occurs in the majority of ALS cases and causes the death of motor neurons via a Ca²⁺ -permeable AMPA receptor-mediated mechanism. Here, we explored the possibility of gene therapy for ALS by upregulating ADAR2 in mouse motor neurons using an adeno-associated virus serotype 9 (AAV9) vector that provides gene delivery to a wide array of central neurons after peripheral administration. A single intravenous injection of AAV9-ADAR2 in conditional ADAR2 knockout mice (AR2), which comprise a mechanistic mouse model of sporadic ALS, caused expression of exogenous ADAR2 in the central neurons and effectively prevented progressive motor dysfunction. Notably, AAV9-ADAR2 rescued the motor neurons of AR2 mice from death by normalizing TDP-43 expression. This AAV9-mediated ADAR2 gene delivery may therefore enable the development of a gene therapy for ALS.
© 2013 The Authors. Published by John Wiley and Sons, Ltd on behalf of EMBO