Now, UAE doctors may be able to treat genetic diseases while baby is in mother's womb

Khaleej Times

28-01-2025

There's new hope for couples who have discovered a genetic disease that may affect their future children. Doctors in the UAE are pioneering new methods that will allow them to treat a genetic disorder while the fetus is still in the mother's womb.
Emirates Health Services (EHS) is bringing in-utero gene editing techniques into the country. This method focuses on directly and permanently correcting gene mutations by altering genes at the DNA level — particularly within the liver — either during pregnancy or shortly after birth.
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'This innovative approach enables the identification of several genetic disorders, including sickle cell disease (thalassaemia), muscular dystrophies, and metabolic abnormalities,' Dr Noora Jasim, paediatric specialist at Al Qassimi Women's and Children's Hospital, told Khaleej Times at the ongoing Arab Health 2025 in Dubai on Tuesday.
The unique characteristics of the fetus provide several advantages for this approach, she said. First, its small size allows for the delivery of "a maximum therapeutic dose of gene editing". Second, the fetus' immunological immaturity reduces the risk of rejection. Third, the target cells to be edited are more plentiful in a fetus than after birth, as they will continue to multiply throughout the individual's life.
How it works
The first step is early genetic screening, a procedure undertaken before marriage or during pregnancy to identify potential genetic diseases that could affect embryos. This process involves conducting genetic tests on parents to detect if there is a genetic disorder.
Dr Noora explained that if a genetic disorder is identified in the parents, a sample can be taken from the mother's blood or through the umbilical cord when the mother is in the second or third trimester of pregnancy (around 16 weeks). The gene responsible for the disease is then modified through gene therapy techniques. This involves targeting and editing specific genes.
Once the gene is modified, it is reintroduced into the body via the umbilical cord, where the edited gene targets the affected tissues before the child is born.
'For example, If parents have a hereditary muscle disease, the gene responsible for this condition can be modified. After editing, the gene is reintroduced through the umbilical cord, specifically targeting the affected muscle tissue before the child's full development," Dr Noora said.
'It is expected that when the child is born, he or she will hopefully not have the hereditary disease."
This screening process is becoming increasingly feasible for addressing additional hereditary genetic disorders. Research is currently exploring the potential to treat Down syndrome, with 407 associated genes identified as potential targets for future therapeutic interventions. This evolving landscape of genetic medicine promises to transform many lives, paving the way for more effective treatments and improved outcomes.
Dr Noora clarified: 'So far, there are no clinical trials on humans for gene therapy. All current research is focused on animal trials, which have been conducted for four years now. While there is a success rate in these trials, gene therapy has not yet been applied to humans.'