Gene Therapy Flowchart PDF

Summary

This document is a flowchart explaining the process of gene therapy. It details the steps involved in identifying the target gene, selecting a vector, constructing the gene, delivering it, monitoring its expression and evaluating its safety and efficacy.

Full Transcript

Gene Therapy Flowchart Gene therapy is a technique that involves introducing genetic material (DNA or RNA) into cells to treat or prevent disease. Here's a simplified flowchart outlining the general process: 1. Identification of the Target Gene Disease-causing mutation: Pinpointing the specific...

Gene Therapy Flowchart Gene therapy is a technique that involves introducing genetic material (DNA or RNA) into cells to treat or prevent disease. Here's a simplified flowchart outlining the general process: 1. Identification of the Target Gene Disease-causing mutation: Pinpointing the specific gene responsible for the disease. Gene function: Understanding the normal role of the gene in the body. 2. Vector Selection Delivery vehicle: Choosing a suitable method to transport the therapeutic gene into cells. Viral vectors: Commonly used, such as adenoviruses, retroviruses, and lentiviruses. Non-viral vectors: Include liposomes, nanoparticles, and physical methods (e.g., electroporation). 3. Gene Construction Therapeutic gene: Creating a functional copy of the gene or a modified version. Regulatory elements: Adding sequences that control when and where the gene is expressed. 4. Gene Delivery In vivo: Directly introducing the gene into the patient's body. Ex vivo: Removing cells from the patient, modifying them, and reintroducing them. 5. Gene Expression Cellular machinery: The introduced gene is transcribed into mRNA and then translated into protein. Therapeutic effect: The protein corrects the genetic defect or provides a new function. 6. Monitoring and Evaluation Safety and efficacy: Assessing the effectiveness of the therapy and any potential side effects. Long-term outcomes: Tracking the patient's health over time. Note: The specific steps and techniques can vary depending on the disease being treated and the type of gene therapy being used. Would you like to know more about specific types of gene therapy or the challenges and limitations associated with this technology?

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