ALS Gene Therapy Insights with Prof. Dame Pamela Shaw
In recent years, genetic therapies have garnered much attention for their potential to modify the underlying pathological processes in neurological diseases. By correcting, replacing, or removing aberrant genetic material, gene therapies could revolutionize the treatment of many diseases, where symptomatic management is the only available option.
Examples of gene therapy methods include the use of CRISPR/Cas9 technologies to directly edit genetic mutations, vector-based gene therapy to deliver new genetic material, and antisense oligonucleotides (ASOs) to modulate gene expression.
The success…
The skinny
The skinny isn't ready yet — notes appear once the transcript is processed.