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Gene therapy: basics and therapeutic applications

Published on April 30, 2023   29 min

A selection of talks on Genetics & Epigenetics

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0:00
Hello everyone. I am Dr. Amit Khurana, a Senior Researcher at the Institute of Molecular Pathobiochemistry, Experimental Gene Therapy, and Clinical Chemistry, at the University Hospital RWTH Aachen, Aachen Germany. I have a PhD in Pharmacology and Toxicology, and my primary research area is the use of novel technologies, including gene therapy for the treatment of chronic inflammation and fibrosis driven diseases. In this lecture, I will introduce you to some of the important basic concepts of gene therapy and its therapeutic applications.
0:43
In this lecture, I will be covering first the introduction to gene therapy, followed by understanding some of the basic concepts of gene therapy. Next, I will provide the information about the various methods of gene transfer, then we will study what is ex vivo and in vivo gene transfer in the context of gene therapy. Finally, we will learn about some of the areas of clinical applications of gene therapy.
1:14
Now, the important question comes. What is gene therapy? To define it, gene therapy is the medical field which focuses on the genetic modification of cells to produce a therapeutic effect. Or it involves the treatment of disease by repairing or reconstructing defective genetic material. In a nutshell, gene therapy essentially deals with manipulating the genetic material that is deoxyribonucleic acid, DNA by using different approaches.

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Gene therapy: basics and therapeutic applications

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