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Showing 1–6 of 6 results

Advanced filters: Author: Atsushi Miyanohara Clear advanced filters

Spinal subpial delivery of AAV9 enables widespread gene silencing and blocks motoneuron degeneration in ALS

Injection of AAV–shRNA below the pial surface of the spinal cord prevents onset or ameliorates progression in a mouse model of ALS, and achieves widespread delivery to the spinal cord and brain motor centers in adult pigs and non-human primates.

Mariana Bravo-Hernandez
Takahiro Tadokoro
Martin Marsala
Research23 Dec 2019
Nature Medicine

Volume: 26, P: 118-130
The sustained expression of Cas9 targeting toxic RNAs reverses disease phenotypes in mouse models of myotonic dystrophy type 1

Molecular and physiological phenotypes in mouse models of myotonic dystrophy type I can be sustainably reversed by CRISPR-mediated targeting of toxic RNA repeats.

Ranjan Batra
David A. Nelles
Gene W. Yeo
Research14 Sept 2020
Nature Biomedical Engineering

Volume: 5, P: 157-168
Viral vectors do the DNA shuffle

Atsushi Miyanohara
Theodore Friedmann
News & Views01 Dec 2000
Nature Biotechnology

Volume: 18, P: 1244-1245
Human mutant huntingtin disrupts vocal learning in transgenic songbirds

The study of speech or vocal disorder resulting from neurological diseases lacks a model capable of recapitulating vocal learning. This study suggests that the vocal disorder associated with Huntington's disease is observed in transgenic zebra finches carrying the full-length human mutant huntingtin gene.

Wan-chun Liu
Jessica Kohn
Ramee Lee
Research05 Oct 2015
Nature Neuroscience

Volume: 18, P: 1617-1622
Bile-duct proliferation as an unexpected side-effect after AAV2-LDLR gene transfer to rabbit liver

Elisa Hytönen
Anniina Laurema
Seppo Ylä-Herttuala
ResearchOpen Access06 May 2019
Scientific Reports

Volume: 9, P: 1-13
The prevalence and clinical features of MYO7A-related hearing loss including DFNA11, DFNB2 and USH1B

Kizuki Watanabe
Shin-ya Nishio
Shunsuke Kondo
ResearchOpen Access09 Apr 2024
Scientific Reports

Volume: 14, P: 1-19