The first clinica

Gene therapy means the correction of defect gene diagnosed in a child or embryo.

It is used to treat defects in somatic(body) or gametic(sperm/egg) cells.

M. Please and W. F. Andresco Attempted gene therapy on a 5-year-old girl who is suffering from adenosine deaminase deficiency.

ADA caused due to the deletion of adenosine deaminase gene.

It can be cured by bone marrow transplantation. But this method is not fully curative.

So we grow lymphocytes from patient's blood in a culture.

Functional ADA DNA was introduced in the lymphocytes with the help of retroviral vector.

Then these Lymphocytes were transferred into the patient's body.

Diseases can be cured by gene therapy are—

i. Cystic fibrosis

ii. Cancer

iii. Parkinson's diseases, etc.

Type of gene therapy are—

i. Somatic gene therapy

In Somatic gene therapy, the Defective gene is replaced by therapeutic genes in somatic cells. The change is not passed on to the next generation.

ii. Germ line gene therapy

In this, germ cells are modified or corrected by introducing functional genes into their genomes. The changes are passed to the next generation.