Unlocking the Potential of Gene Therapy for Severe Diseases

🔬Genes are segments of DNA that direct the cell to produce specific proteins, playing a vital role in the body's functions.

⚙️Gene therapy delivers a functional copy of the gene into the patient's cells, allowing the production of a normal protein and correcting the underlying cause of the disease.

🧬AAV vectors, engineered viruses, are well-suited for delivering genes into cells, particularly in treating retinal diseases.

👁️Inherited retinal diseases, such as achromatopsia, can cause vision loss and complete color blindness.

💡Gene therapy has the potential to provide long-lasting clinical benefits by introducing a functional gene that remains stable for many years.

What is gene therapy?

—Gene therapy is a treatment that delivers a functional gene into a patient's cells to correct the underlying genetic defect causing a disease.

How does gene therapy work?

—Gene therapy uses engineered viruses called AAV vectors to deliver the functional gene. Once inside the patient's cells, the functional gene supports the production of a normal protein, correcting the disease's underlying cause.

What diseases can gene therapy treat?

—Gene therapy shows potential in treating a wide range of diseases with a genetic basis, including inherited retinal diseases, muscular dystrophy, and certain types of cancer.

Are there any side effects or risks associated with gene therapy?

—While gene therapy holds promise, there can be risks and side effects. These include immune responses to the viral vectors, potential insertional mutagenesis, or off-target effects.

Is gene therapy a permanent solution?

—Gene therapy has the potential to provide long-lasting clinical benefits, as the functional gene delivered by the AAV vectors can remain stable for many years. However, additional studies and follow-up assessments are necessary to fully understand the duration of the therapeutic effects.

00:00Gene therapy is using engineered viruses to develop long-lasting therapies for severe diseases, focusing on ophthalmology and genetic defects in over 200 known genes.

00:33Genes are segments of DNA that provide instructions for producing proteins and can be associated with various diseases.

01:17In gene therapy, functional genes are delivered to the patient's cells using adeno-associated viruses (AAV), which have shown efficacy and safety in retinal diseases.

02:01Achromatopsia, an inherited retinal disease, causes severe visual impairments, including reduced acuity, light sensitivity, and color blindness.

03:34Gene therapy aims to restore the function of cone photoreceptors in the retina by introducing a functional copy of the defective genes.

03:59Gene therapy has the potential for long-lasting clinical benefits, and A GTC specializes in developing gene therapies for rare inherited retinal diseases.

04:16For more information about gene therapy and A GTC's work, visit their website at www.gtc.com