Gene Therapy

New treatments, new hope

Thousands of diseases are caused by a single genetic defect. Until recently, most of these disorders had ineffective treatments available, or no treatment at all. Scientific advances like gene therapy have opened a world of treatment options for patients with genetic disorders worldwide.

Gene therapy replaces broken genes with normal functional genes, allowing a patient’s own body to produce proteins to treat their illness. A single treatment provides long-lasting benefit – sometimes even for a lifetime – leading to a better quality of life for patients worldwide.

The most common form of gene therapy involves inserting normal, healthy genes into the patient so that the healthy gene can replace or supplement the activity of a damaged gene. To ensure that the functional gene is delivered inside cells where the protein can be made, it is carried by a ‘vector’ that can enter the cell without harming it. Vector systems that are derived from viruses for use in gene delivery are generally considered the most efficient.

Gene therapy is becoming a reality, as evidenced by positive clinical data, the first regulatory approval of a gene therapy and increasing industry investments. Data in multiple rare and severe genetic diseases shows that gene therapy is no longer just a promise, but represents the emergence of a new therapeutic modality for patients with diseases that today have limited or no therapeutic alternatives.

What is a Gene?

A gene is an important unit of hereditary information in a living organism, as it provides the code for a living organism’s traits, characteristics, and physical development. Humans have about 20,000 genes that are located on 46 chromosomes. Gene therapy corrects genetic errors that lead to malfunctioning or even absent proteins, which are at the root of many diseases. Proteins are involved in many processes within cells and act as building blocks, critical in the “communication” among cells.

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AGTC
11801 Research Drive, Suite D
Alachua, Florida 32615
386.462.2204
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