Advances in gene therapy are changing the way we treat genetic disorders, giving people new options for treatment and hope for cures.

1. What Are Genetic Disorders?
   Genetic disorders are conditions that affect about 1 in 10 people. They can be divided into two main types:

   • Single-gene disorders: These are caused by problems in one specific gene. Examples include cystic fibrosis and sickle cell disease.
   • Complex genetic disorders: These involve multiple genes and can be influenced by lifestyle and environment. Examples include diabetes and heart disease.

2. How Does Gene Therapy Work?
   There are some exciting new techniques in gene therapy:

   • CRISPR-Cas9: This cutting-edge technique allows scientists to edit genes very precisely. It has shown promise in early tests for conditions like muscular dystrophy.
   • Adeno-Associated Viral Vectors (AAV): These are like tiny delivery trucks that transport helpful genes to the right cells in the body. A great example is Zolgensma, which is used for treating spinal muscular atrophy. However, it is very expensive, costing around $2.1 million per patient.

3. How Successful Is Gene Therapy?
   Clinical trials have shown impressive results:

   • For sickle cell disease, CRISPR therapy has helped more than 80% of patients get closer to a cure in early studies.
   • The use of gene therapy has increased the 5-year survival rate for some inherited disorders. For example, it improved from about 40% to nearly 90%.

4. Government Approval
   The FDA has created a quicker approval process for gene therapies, allowing patients to access them sooner. In 2022, more than 15 gene therapies were approved, showing that this new approach is being accepted more widely.

In summary, advances in gene therapy are changing how we treat genetic disorders. They are improving outcomes for patients and bringing hope to those with conditions that were once hard to treat. As research continues, we can expect even more exciting developments in how we manage genetic and congenital disorders, leading to better care for patients.