Our commitment to rare and life-threatening NEUROLOGICAL genetic diseases

We work relentlessly to bring gene therapy to patients and families affected by rare and life-threatening neurological genetic diseases, like spinal muscular atrophy (SMA), Rett syndrome (RTT), and a genetic form of amyotrophic lateral sclerosis (ALS). Our dedication to advancing gene-based medicine can make a difference in the fight against devastating hereditary diseases.

HOW GENE THERAPY WORKS

When exploring different treatment options for your loved one, it's important to understand how gene therapy works. Gene therapy addresses the root cause of a genetic disease by replacing the function of a missing or faulty gene with a new working copy of the gene in order to restore the function of the protein.

The major steps of gene therapy are:

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Step 1The vector—an altered adeno-associated virus that can't reproduce—is used to deliver a new gene. The vector binds to the patient's cell membrane.

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Step 2The vector breaks down, allowing the new gene to be injected into the cell nucleus.

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Step 3With the new gene in place, the patient's cell begins producing the required protein.

Visit www.exploregenetherapy.com for more information.

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