Gene Therapy: Mechanism of Gene therapy

Gene therapy is an amazing technology to edit genes and treat genetic disorders.
Main goals of gene therapy
1) To replace a faulty or defective gene with normal or Wild Gene.
2) Make faulty gene silent
3) Introduce new gene
Limitations and challenges with gene therapy
1) Its hard to target specific cells
2) You can edit only Monogenic Disorders
3) Gene therapy can disrupt normal genes
4) Gene therapy can trigger Immune response
5) Its hard to treat large genes in gene therapy
Procedure of gene therapy
There are two ways by which gene therapy is performed
1) Ex-vivo Gene therapy
Ex vivo gene therapy is a targeted approach where cells are removed from the patient's body, genetically modified in a controlled environment, and then reintroduced into the patient. This technique allows for precise gene editing and correction before the cells are returned to combat genetic disorders. It's like a genetic pit stop to fix the glitches before rejoining the race of healthy cellular function.
2) In-Vivo gene therapy
In vivo gene therapy involves directly delivering genetic material into the patient's body to treat diseases. It's like performing genetic "surgery" within the patient, addressing the genetic issue at its source without removing or modifying cells externally. This approach offers potential treatments for a wide range of conditions by targeting the specific tissues or organs affected.
Vectors in Gene therapy
Vectors in gene therapy are like molecular delivery vehicles. They carry the therapeutic genetic material to target cells. Often, modified viruses serve as vectors, acting as safe carriers to transport and deliver the genetic cargo precisely where it's needed for effective gene therapy. Think of them as genetic couriers, ensuring the therapeutic payload reaches its destination.
Example of Gene Therapy
Cystic Fibrosis
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