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ASO Treatment

Snapshot: What are single nucleotide polymorphisms (SNPs)?

It’s in our DNA If you were to unravel the tightly wound packages of our genome known as chromosomes, you would find long strings of DNA. The strings are made up of only four different building blocks, compounds abbreviated as adenine (A), thymine (T), guanine (G) and cytosine (C). Picture Read More…

Snapshot: What is Gene Therapy?

Gene therapy is using nucleic acids to treat a genetic disorder.  These nucleic acids can be designed in a variety of ways to achieve the same therapeutic outcome. Gene therapy tools can be used to correct a mutant gene by one of three ways: Expressing a healthy copy of a Read More…

Snapshot: What is an antisense oligonucleotide (ASO/AON)?

Antisense Oligonucleotides (also known as ASOs or AONs) are small molecules that can be used to prevent or alter the production of proteins. Proteins are the workforce of the cell, taking care of most cellular processes. They are generally made in a two-step process: first, a specific protein-coding gene is Read More…

Approaching the age of clinical therapy for spinocerebellar ataxia type 1

Written by Dr. Marija Cvetanovic Edited by Dr. Maxime W. Rousseaux New research (published Nov. 2018) reveals promising potential genetic therapy for SCA1. A research team comprised of scientists from academia and industry have tested a new treatment for Spinocerebellar ataxia type 1 (SCA1), bringing disease-modifying therapy one step closer Read More…

A novel therapeutic approach for the treatment of SCA3

Written by Larissa Nitschke Edited by Dr. Gülin Öz Researchers in the Netherlands uncover a new way to treat SCA3 Upon receiving a conclusive diagnosis of Spinocerebellar Ataxia (SCA), hundreds of questions can appear in a patient’s mind: What is Spinocerebellar Ataxia? Why am I affected? How will my symptoms Read More…

ASOs clear toxic protein from cells, reducing ataxia in SCA2 mice

Written by Anna Cook and Dr. Alanna Watt Edited by Dr. Vitaliy V. Bondar Scientists uncover a promising therapeutic avenue to treat spinocerebellar ataxia type 2 (SCA2). Spinocerebellar ataxia type 2 (SCA2) is a progressive ataxia caused by a mutation in the ATXN2 gene. This mutation causes a tract of the amino Read More…

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