Repeat Associated Diseases
Repeat associated diseases are a group of genetic diseases, also known as Trinucleotide expansion diseases. Some of the most challenging diseases belong to this group such as Huntington’s disease, Amyotrophic Lateral Sclerosis (ALS), Fragile X tremor ataxia and Myotonic dystrophy type 1/2.
At Anima, we believe that the malfunction of the translation machinery is the common intervention point to find therapeutics for those devastating and yet untreatable diseases. Our unique approach allows us to visualize the different types of translation malfunctions and enables novel treatments.
We are in the process of running discovery programs around several diseases in this area.
|Fragile X tremor ataxia||non-coding||FMR1|
|Spinobulbar muscular atrophy||coding||AR|
|Spinocerebellar Ataxias (SCA types 1, 2, 3, 6, 7, 17)||coding||ATXN-1, 2, 3, 7, CACNA1A, TBP|
|Dentatorubro-Pallidoluysian Atrophy (DRPLA)||coding||ATN1|
|Myotonic dystrophy type 1||coding||DMPK|
|Myotonic dystrophy type 2||coding||CNBP|
|Spinocerebellar Ataxia, type 10||coding||Ataxin10|