In the future When gene therapy can precisely tune a person’s genome to cure a serious disease, early treatment is often better. And the uterus will recover as soon as possible. At the annual meeting of the American Society for Gene and Cell Therapy in Los Angeles last week, several researchers presented advances in in utero gene therapy, and studies in experimental animals offer some hope for human treatments. However, it indicated that it still faces significant challenges. hurdle.
Doctors can already detect abnormalities in the DNA of developing human fetuses. Conditions such as sickle cell anemia and spinal muscular atrophy are caused by genetic features (sometimes as simple as a single genetic mutation) that show up in prenatal screening. Newer gene therapies can treat adults and even children with these diseases, but they have some drawbacks. A single dose can cost millions of dollars, and many are currently only available to participants in clinical trials. Best of all, by the time the person receives them, they may already be living with a serious illness for months, if not years.
Doctors and scientists hope that correcting these abnormalities before birth will increase the chances of a healthy life for the newborn. “The main advantage of administering these treatments in utero or prenatally is that they prevent disease before it occurs,” said Bill Peranteau, a pediatric and fetal surgeon at Philadelphia Children’s Hospital, the team presented at the meeting. says Mr.
His team has studied many diseases, including cystic fibrosis and metabolic disorders. Other conference attendees presented studies on conditions affecting the liver, muscles, brain and spinal cord. Overall, these teams tend to focus on serious diseases that would benefit from early treatment. For some diseases, “waiting until after birth, or too long after birth, is a missed opportunity to prevent irreversible disease,” Peranteau said.
Fetal development provides that window because at that point it has many undifferentiated stem cells that give rise to all kinds of body tissues. Because the fetus’s immune system is not fully mature, it is less likely to reject biologic therapy than the immune system of an infant. And because it’s so small, it also requires less complex and expensive drug dosages.
A gene therapy “drug” is not a drug in the traditional sense. It is not a compound that kills invading bacteria or calms the symptoms of disease. Instead, DNA-editing tools (such as Crispr) are often packaged in some kind of delivery vehicle (usually a virus or nanoparticle) that carries the DNA to target cells. In this therapy, genes can be cut, replaced, or inserted to reverse or alleviate disease.
“Birth is still a huge challenge,” says Rohan Palanchi, a bioengineer and Ph.D. This is because it is difficult to target cells in organs such as the brain, lungs and skin. The best way to solve fertility problems depends on the type of disease and gene therapy. Researchers may be able to optimize the microscopic vehicle that delivers the genetic machinery, inject drugs at specific regions or times during pregnancy, or do all of the above.