TANGO Gene Therapy Could Treat Dravet Syndrome

What do medication, vagus nerve stimulation, and low-carb ketogenic diets all have in common? Currently, these are all therapeutic options for those with Dravet syndrome. However, researchers and doctors believe that additional therapies are needed – both to improve patient outcomes and to address the underlying cause of this rare epilepsy. According to Medical XPress, a research team from the University of Virginia School of Medicine has discovered that TANGO gene therapy could benefit patients and improve the overall quality of life. 

Interested in learning more? Take a look at the findings, published in Brain Research, to learn how TANGO gene therapy impacted mice models of Dravet syndrome. 

TANGO Gene Therapy

Developed by biotechnology company Stoke Therapeutics, TANGO (which stands for Targeted Augmentation of Nuclear Gene Output) is:

Stoke’s proprietary research platform [which designs] antisense oligonucleotides that bind to pre-mRNA and help the target genes produce more protein. TANGO aims to restore missing proteins by increasing – or stoking – protein output from healthy genes, thus compensating for the non-functioning copy of the gene.

For example, SCN1A mutations typically cause Dravet syndrome. These mutations prevent the production of enough SCN1A protein. TANGO gene therapy aims to address the underlying cause of Dravet syndrome by prompting increased SCN1A production. 

In this particular research, the researchers evaluated TANGO gene therapy in mice models of Dravet Syndrome. To begin, researchers treated infant mice with a singular TANGO treatment. Findings included:

  • TANGO gene therapy prevented seizures and early mortality in infant mice.
  • Treatment prompted interneurons to believe that there was an adequate amount of SCN1A protein. Ultimately, this helped the cells to function properly and actively, reducing seizure frequency and severity.

About Dravet Syndrome

An estimated 1 in every 15,700-40,000 live births is affected by Dravet syndrome, a rare genetic epileptic encephalopathy. This form of treatment-averse epilepsy usually manifests within the first year following birth and lasts for a lifetime. Dravet syndrome is characterized by prolonged febrile and non-febrile seizures in the first year, followed by myoclonic and partial seizures, atonic seizures, as well as other forms, in later years. If your child is under one year old and has seizures occurring on one side of the body, seizures lasting for 10+ minutes, or seizures triggered by warm water, consider pursuing a Dravet syndrome diagnosis.

Other potential symptoms associated with Dravet Syndrome include:

  • Psychomotor delay
  • Unsteady gait
  • Developmental delays
  • Impaired coordination
  • Delayed language and speech
  • Behavioral issues
  • Chronic infections
  • Dysautonomia
  • Sudden unexpected death in epilepsy (SUDEP)

Learn more about Dravet syndrome.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

Follow us