Around 43 million people worldwide suffer from blindness. Jean Bennett is a gene therapy expert and professor of ophthalmology at the University of Pennsylvania. He and his lab developed the first gene therapy drug to be approved in the US.
(Conversation) New scientific discoveries have outlined new approaches to treating certain types of genetic blindness through ‘gene therapy’. This has raised hopes of bringing light to the lives of 29.5 crore people suffering from vision impairment globally. Around 43 million people worldwide suffer from blindness. Jean Bennett is a gene therapy expert and professor of ophthalmology at the University of Pennsylvania. He and his lab developed the first gene therapy drug to be approved in the US.
The drug ‘Luxturna’ is used to treat patients suffering from vision impairment and loss of vision due to a rare genetic disease early in life. In patients with this disease, biallelic RPE65 mutations cause retinal damage. Bennett answered in detail the questions asked by ‘Conversation’ about this, the main parts of which are – What is gene therapy and how does it work? Gene therapy is a group of techniques that use DNA or RNA to treat or prevent disease.
Gene therapy treats disease in three primary ways—replacing the disease-causing gene with a healthy and new or modified copy of that gene, activating or deactivating the gene, and introducing a new or modified gene into the body. Gene entry. How do doctors treat genetic eye diseases and blindness? In the past, many physicians did not consider it necessary to characterize the genetic basis of eye disease because a cure was not available. However, some experts (my colleagues and I included) have characterized these genetic aberrations in their research and are convinced that a cure will someday be possible.
In due course of time Im was able to develop a method of treatment especially for those who suffered from congenital blindness due to a specific genetic defect. The development of gene therapy for genetic disease has also inspired other groups around the world to conduct ‘clinical’ trials targeting other genetic forms of blindness. Genetic factors lead to diseases like ‘choroideremia’, ‘achromatopsia’, ‘retinitis pigmentosa’ and even age-related ‘macular damage’ which leads to loss of vision.
There are at least 40 ongoing ‘clinical trials’ on several genetic variants that cause blindness. You developed one of the first gene therapies to be approved in the US, but what is the current status of clinical use of gene therapy? There are several gene therapies now approved in the US, but most are combined with cell therapy. Under this, a cell is first modified and it is reintroduced into the patient’s body.
In a new injectable gene therapy, a drug called ‘Skysona’ is given to children between the ages of four and 17 with a genetic disease of the brain (adrenoleukodystrophy). More than two dozen cell and gene therapies have been approved by the US body FDA, which includes CAR T-cell therapy. In ‘CAR T-cell therapy’, T cells (a type of cells related to the immune system) are modified so that they better attack cancer cells in the body.
What is the biggest challenge facing gene therapy? The biggest challenges include systemic diseases, or diseases that affect the whole body rather than a single organ or part of the body. High-dose gene therapy should be given for such diseases, but the challenge is how to prepare large enough quantities of the gene therapy compound to treat the targeted diseased tissue without causing any side effects.
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