Back to results
US 2019/0167815 A1Published application
Methods and compositions for the treatment of rare diseases
OwnerMichael C. Holmes, Brigit E. Riley +3inventors
InventorsMichael C. Holmes, Brigit E. Riley, Thomas Wechsler, Bryan Zeitler, Lei Zhang
Life of this patent
Estimating the term…
Abstract
The present disclosure is in the field of modulation of genes involved in rare diseases including for diagnostics and therapeutics for rare diseases such as Angelman's Syndrome, Facioscapulohumeral Muscular Dystrophy (FHMD), Amyotrophic Lateral Sclerosis (ALS), Frontotemporal dementia (FTD) and Spinal Muscular Atrophy (SMA).
Claims17
A genetic modulator of a C9orf72 gene, the modulator comprising
- a DNA-binding domain that binds to a target site of at least 12 nucleotides in the C9orf72 gene;
- and
- a transcriptional regulatory domain or nuclease domain.
Dependencies are inferred from the claim text.