How can genetic disorders be cured

Your health care provider can then give you individualized and specific education about how to: Check regularly for the disease. Follow a healthy diet. Get regular exercise. Avoid smoking tobacco and too much alcohol. Get specific genetic testing that can help with diagnosis and treatment. How do I find a genetic professional? Can knowing about genetics help treat disease? How is genetic information used to treat disease? What are some concerns? These concerns include: Tailor-made medicines might be more expensive Not everyone might have access to new treatments Keeping genetic information private Possible discrimination at work and from health insurance companies Need for more information about this type of medicine.

But several significant barriers stand in the way of gene therapy becoming a reliable form of treatment, including:. Gene therapy continues to be a very important and active area of research aimed at developing new, effective treatments for a variety of diseases. Explore Mayo Clinic studies of tests and procedures to help prevent, detect, treat or manage conditions. Mayo Clinic does not endorse companies or products. Advertising revenue supports our not-for-profit mission. This content does not have an English version.

This content does not have an Arabic version. Overview Gene therapy involves altering the genes inside your body's cells in an effort to treat or stop disease. Request an Appointment at Mayo Clinic. Share on: Facebook Twitter. Show references Gene therapy. Genetics Home Reference. Accessed July 21, Conditions that are characterized by defective blood cell formation, such as sickle cell disease , can sometimes be treated with a bone marrow transplant.

Bone marrow transplantation can allow the formation of normal blood cells and, if done early in life, may help prevent episodes of pain and other future complications. Some genetic changes are associated with an increased risk of future health problems, such as certain forms of cancer. Management may include more frequent cancer screening or preventive prophylactic surgery to remove the tissues at highest risk of becoming cancerous. Genetic disorders may cause such severe health problems that they are incompatible with life.

In the most severe cases, these conditions may cause a miscarriage of an affected embryo or fetus. In other cases, affected infants may be stillborn or die shortly after birth. Although few treatments are available for these severe genetic conditions, health professionals can often provide supportive care, such as pain relief or mechanical breathing assistance, to the affected individual.

Here is a list of some of the first diseases that scientists are tackling using CRISPR-Cas technology, testing its possibilities and limits as a medical tool. The gene-editing technology is used to remove the gene that encodes for a protein called PD This protein found on the surface of immune cells is the target of some cancer drugs such as checkpoint inhibitors.

This is because some tumor cells are able to bind to the PD-1 protein to block the immune response against cancer. The trial tested this approach in 12 patients with non-small cell lung cancer at the West China Hospital. The results, published in April , suggested the approach was feasible and safe.

Some experts have recommended that the long-term safety of the approach remain under review. Others have suggested using more precise gene-editing approaches such as base editing. In the US, a phase I trial run by the University of Pennsylvania tested the safety of a similar approach. They then added another gene to help the immune cells recognize tumors.

T he results revealed that the treatment was safe in patients with advanced forms of cancer. The therapy consists of harvesting bone marrow stem cells from the patients and using CRISPR technology in vitro to make them produce fetal hemoglobin. This is a natural form of the oxygen-carrying protein that binds oxygen much better than the conventional adult form.

The modified cells are then reinfused into the patient.