October 2018

SLC-Square

FOR IMMEDIATE RELEASE

Contact: Amber Freed, SLC6A1 Connect
Phone: 303-907-8038
Email: afreed@slc6a1connect.org

SLC6A1 Connect Partners with Dr. Steven Gray

Pre-clinical lab work currently underway on gene replacement therapy to treat SLC6A1

DENVER, CO, October 2018 – SLC6A1 Connect is proud to announce a partnership with Dr. Steven Gray from UT Southwestern to develop a gene replacement therapy to treat SLC6A1 mutation. Pre-clinical lab work is currently underway.

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Article: Patients with rare brain diseases help scientists open new doors for gene therapy

Why Gene Therapy?

Scientific Explanation: SLC6A1 is a heterozygous haplo-insufficient, loss of function and less than 6kb in size so the gene fits nicely into AAV9.

Patient Explanation: Despite the eligibility hurdles in place for gene therapy, SLC6A1 meets the criteria for a good fit.

How does Gene Therapy Work?

The explanation is very simple but the process is very complex. Simply stated, a good copy of the broken DNA (from anyone) is inserted into a vector (a harmless virus called AAV9) that carries the good DNA to the brain. The virus then stacks good copies of the DNA on top of the bad copies of the DNA and the pathway is opened.

SLC6A1 Connect is a patient advocacy group dedicated to improving the lives of children and families affected by SLC6A1. Our focus is to raise awareness and funding to advance scientific research that will ultimately result in a cure.

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If you would like more information about this topic, please contact Amber Freed at 303-907-8038, or e-mail afreed@slc6a1connect.org