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Gene Therapy Introduction

How does gene therapy work?

  • Genes are composed of Deoxyribonucleic acid – DNA. DNA is composed of genetic information, by which they produce the specific proteins which are the building blocks of the body. 
  • Gene damage or missing gene may lead to inadequate or wrong protein production leading to disease condition.
  • In gene therapy, a normal gene is inserted into the genome to replace an abnormal disease causing gene.
  • A carrier molecule “vector” can be used to deliver the therapeutic gene into the patients target cells.
  • Viruses are microorganisms which infect humans and multiply in the genome of the human body.
  • Scientists have tried to take advantage of this multiplication capacity of the viruses to transfer the required genes into the human body for treatment purposes.
  • The virus is manipulated by the scientists and the pathogenic disease causing genes in the virus are removed and the therapeutic genes are inserted.
  • The virus vector then unloads its genetic material containing the therapeutic human gene into the target cell (human host).
  • The treated human produce the normal proteins due to the transfer of the therapeutic genes.

Related Links
Gene Therapy Treatment
Genetic Counseling