Therapeutic Gene Silencing Delivered by a Chemically Modified Small Interfering RNA against Mutant SOD1 Slows Amyotrophic Lateral Sclerosis Progression*♦
H Wang, A Ghosh, H Baigude, C Yang, L Qiu… - Journal of biological …, 2008 - ASBMB
Inherited neurodegenerative diseases, such as Huntington disease and subset of Alzheimer
disease, Parkinson disease, and amyotrophic lateral sclerosis, are caused by the mutant
genes that have gained undefined properties that harm cells in the nervous system, causing
neurodegeneration and clinical phenotypes. Lowering the mutant gene expression is
predicted to slow the disease progression and produce clinical benefit. Administration of
small interfering RNA (siRNA) can silence specific genes. However, long term delivery of …
disease, Parkinson disease, and amyotrophic lateral sclerosis, are caused by the mutant
genes that have gained undefined properties that harm cells in the nervous system, causing
neurodegeneration and clinical phenotypes. Lowering the mutant gene expression is
predicted to slow the disease progression and produce clinical benefit. Administration of
small interfering RNA (siRNA) can silence specific genes. However, long term delivery of …
