News Health/Medical Experimental Gene Therapy Shows Promise for Giant Axonal Neuropathy
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Giant axonal neuropathy

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Experimental Gene Therapy for Giant Axonal Neuropathy Shows Promise in NIH Clinical Trial

An investigational gene therapy for a rare neurodegenerative disease known as giant axonal neuropathy (GAN) has shown promise in a clinical trial conducted by the National Institutes of Health (NIH). GAN typically begins in early childhood and lacks effective treatments, resulting in a fatal outcome by the age of 30.

Therapeutic Breakthrough

Fourteen children, aged 6 to 14 years, participated in the trial at the NIH Clinical Center. The gene therapy, administered directly into the spinal fluid, utilizes a modified virus to deliver functional copies of the defective GAN gene to nerve cells. Notably, this marks the first time a sensory nerve affected by a genetic degenerative disease has shown signs of recovery through gene therapy.

Promising Results

Over a six-year follow-up period, the treatment demonstrated favorable safety and tolerability. Although GAN remains a rapidly progressive disease, the therapy appeared to slow the rate of motor function decline. Additionally, there were indications of sensory nerve regeneration in some patients.

“Waking Up” Sensory Nerves

Dr. Carsten G. Bonnemann, senior author of the study, expressed excitement about the sensory nerves “waking up” again in certain patients. The gene therapy, called scAAV9/JeT-GAN, was administered via an injection into the spinal fluid, with minimal adverse effects.

Hope for GAN Patients

These early-stage trial results offer hope for individuals with GAN, suggesting that gene therapy may provide a viable treatment option. Further research and development are needed to refine and expand this promising approach.

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