Genes hold the instructions that shape every cell in the body, directing the production of proteins that allow cells to grow, function, and divide. Gene therapy uses this genetic coding to address or prevent diseases by modifying these instructions within cells.
Defective genes can cause cells to produce dysfunctional proteins or prevent their production altogether, impacting normal body functions. Gene therapy aims to correct these issues, often by targeting the problem at its source. In vivo gene therapy, for example, uses viruses or tools like CRISPR (clustered regularly interspaced short palindromic repeats) within the body to modify cellular DNA directly, addressing genetic abnormalities inside cells.
Approved therapies now include gene transfer methods for high-risk, non-muscle invasive bladder cancer, delivered through an adenovirus-based approach. The FDA also approved gene therapies in 2023 for conditions caused by single-gene mutations, like sickle cell disease, using CRISPR to correct the genetic anomaly.
Researchers have developed other gene therapy techniques, such as ex vivo therapy, where they modify a patient’s immune cells outside the body before re-infusing them. Treatments like CAR T-cell therapy (for specific blood cancers), CAR NK-cell therapy (currently in trials), therapeutic cancer vaccines (like the FDA-approved prostate cancer vaccine), and oncolytic viruses (targeting advanced melanoma) genetically engineer immune cells or viruses to target cancer cells.
Defective genes can cause cells to produce dysfunctional proteins or prevent their production altogether, impacting normal body functions. Gene therapy aims to correct these issues, often by targeting the problem at its source. In vivo gene therapy, for example, uses viruses or tools like CRISPR (clustered regularly interspaced short palindromic repeats) within the body to modify cellular DNA directly, addressing genetic abnormalities inside cells.
Approved therapies now include gene transfer methods for high-risk, non-muscle invasive bladder cancer, delivered through an adenovirus-based approach. The FDA also approved gene therapies in 2023 for conditions caused by single-gene mutations, like sickle cell disease, using CRISPR to correct the genetic anomaly.
Researchers have developed other gene therapy techniques, such as ex vivo therapy, where they modify a patient’s immune cells outside the body before re-infusing them. Treatments like CAR T-cell therapy (for specific blood cancers), CAR NK-cell therapy (currently in trials), therapeutic cancer vaccines (like the FDA-approved prostate cancer vaccine), and oncolytic viruses (targeting advanced melanoma) genetically engineer immune cells or viruses to target cancer cells.
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