Advancements in Gene Therapy
Cell and gene therapies are rapidly advancing fields in medicine, with new breakthroughs being made on a regular basis. Here are some of the current advancements in these areas:
- CAR-T cell therapy: Chimeric antigen receptor T-cell (CAR-T) therapy is a type of immunotherapy that involves modifying a patient’s own T-cells to target cancer cells. In 2021, the US Food and Drug Administration (FDA) approved several new CAR-T therapies for the treatment of different types of cancer, including relapsed or refractory large B-cell lymphoma and multiple myeloma.
- CRISPR gene editing: Clustered regularly interspaced short palindromic repeats (CRISPR) is a revolutionary gene editing technology that allows scientists to modify genes with precision. In 2021, researchers used CRISPR to edit genes in human embryos for the first time to study the development of early human life.
- Gene therapy for inherited diseases: Gene therapy is a technique that involves replacing or repairing faulty genes that cause genetic diseases. In 2021, the FDA approved a gene therapy for the treatment of inherited retinal disease caused by mutations in the RPE65 gene.
- Stem cell therapy: Stem cells have the ability to differentiate into different types of cells in the body, making them useful for regenerative medicine. Researchers are exploring the use of stem cell therapy for a variety of conditions, including heart disease, spinal cord injuries, and diabetes.
- mRNA vaccines: mRNA vaccines are a new type of vaccine that use a small piece of genetic material from the virus to stimulate an immune response. In 2020, mRNA vaccines were developed and approved for emergency use to protect against COVID-19, marking a major milestone in the field of gene therapy.
These are just a few of the many exciting advancements in cell and gene therapies. As research continues in these areas, we can expect to see even more breakthroughs in the coming years.