Regardless of where you stand on gene-editing, the results of clinical trials that use CRISPR gene editing to improve vision and reduce the effects of type 1 diabetes are encouraging. CRISPR is a tool that allows scientists to essentially edit DNA sequences in humans and animals. The main objective is to edit out mutations in the DNA strand that cause disease and disability. Recently, research has focused on vision loss due to genetic conditions such as Leber Congenital Amaurosis, or LCA, and on pancreatic deficiencies in those who suffer from T1D. Learn more about what’s being done to improve both conditions.
CRISPR Technology and Defective Photoreceptors
In patients who have Leber Congenital Amaurosis, a faulty photoreceptor in the eye is to blame for partial or total vision loss. While most people who have LCA aren’t born blind, many lose their sight soon thereafter — usually within the first months of life. This happens because impaired photoreceptors are unable to send sufficient messages to the brain. Because there’s a disconnect between what the eye sees and what the photoreceptor interprets, vision becomes impaired or lost. This condition is not a result of the inability to see, but rather the inability of the brain to translate information into visual images. And it happens because a single photoreceptor gene has a mutation that prevents it from producing the right protein.
Now, imagine what might happen if your eye doctor could replace that faulty gene. There’s a possibility that your vision might be improved or even restored. That’s the beauty of what scientists are currently capable of doing using the magic of CRISPR gene editing.
Right now, clinical trials to improve vision in subjects suffering with LCA involve injecting a viral vector that contains the needed protein directly into the eye. The virus penetrates the cell, giving it access to the needed protein, and the gene editing happens in-vivo, or inside the body. To date, results have been optimistic. Research is still in its earliest stages, however, and much more study is needed to bring this treatment to the forefront as a cure for LCA.
How CRISPR Technology Is Helping Patients with Type 1 Diabetes
Someone who has Type 1 diabetes doesn’t have enough pancreatic beta cells — the cells that produce insulin. As a result, they struggle with high blood-sugar levels all their lives. Often, T1D leads to problems with major organs such as the kidneys, liver, and heart. In some instances, it can cause premature death.
T1D can’t be effectively controlled through lifestyle changes alone, which is why it’s one of the toughest diseases to manage. However, through the science of CRISPR gene editing, there may be new hope for patients who are born with this disabling condition.
In previous attempts at immunotherapy to replace defective pancreatic cells, patients had to take immunosuppressant drugs to prevent the body from attacking and destroying the implanted cells. However, in newer versions of CRISPR gene therapy, cells have been edited in such a way that the body does not see them as threats. These healthy pancreatic cells are introduced into the body in tiny pouches. Blood vessels grow around the pouches, which makes them appear normal to the body’s internal defense systems. The goal is to provide the patient with enough healthy pancreatic cells that they can produce needed levels of insulin without the need for side-effect-laden immunosuppressant drugs.
CRISPR gene editing has already come a long way in helping to treat pediatric and adult forms of cancer. Now, it’s gaining ground as an effective treatment for both LCA and T1D, too. What could be next for this groundbreaking form of cell therapy? Scientists are excited to find out.