gene #silencer tagged posts

Novel spinal therapy/delivery approach prevented disease onset in neurodegenerative ALS disease model in adult mice and blocked progression in animals already showing disease symptoms.

Writing in Nature Medicine, an international team headed by researchers at University of California San Diego School of Medicine describe a new way to effectively deliver a gene-silencing vector to adult amyotrophic lateral sclerosis (ALS) mice, resulting in long-term suppression of the degenerative motor neuron disorder if treatment vector is delivered prior to disease onset, and blockage of disease progression in adult animals if ...

IMAGE: Huntington’s disease is due to a fault (a repeat of DNA) on chromosome 4. This repetition of genetic information results in the production of an abnormal protein called huntingtin,. Mutant huntingtin aggregates in clusters in the nuclei of certain neurons in the brain, particularly the striatum, a brain structure involved in controlling movement. These neurons degenerate and eventually die, explaining the appearance of abnormal movements. Then huntingtin accumulates in the nuclei of neurons in the cerebral cortex also causing neuronal degeneration with consequent cognitive impairment (memory, attention …) and psychiatric disorders.

A drug that would be the first to target the cause of Huntington’s disease (HD) is effective and safe when tested in mice and monkeys...