What is gene therapy? Illustrate using the example of adenosine deaminase $(ADA)$ deficiency.

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(N/A) Gene therapy is a technique for correcting a defective gene through gene manipulation. It involves the delivery of a normal gene into the individual to replace the defective gene,for example,the introduction of the gene for adenosine deaminase $(ADA)$ in an $ADA$-deficient individual.
The adenosine deaminase enzyme is crucial for the normal functioning of the immune system. Individuals suffering from this disorder can be treated by the transplantation of bone marrow cells.
The process involves the following steps:
$1$. Extraction of lymphocytes from the patient's bone marrow.
$2$. Introduction of a functional $ADA$ gene into these lymphocytes using a retroviral vector.
$3$. Re-introduction of these genetically modified lymphocytes into the patient's bone marrow.
Once inside,the gene gets activated,producing functional $T$-lymphocytes and restoring the patient's immune system.

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Similar Questions

$A$: Recombinant $DNA$ technology is less effective in therapeutic drug production.
$R$: Recombinant therapeutics induce unwanted immunological responses.

Given below is the sequence of events in the production of human insulin by Eli Lilly.
$(a)$ Preparation of two $DNA$ sequences corresponding to chain $A$ and $B$ of human insulin.
$(b)$ Production of chain $A$ and $B$ separately.
$(c)$ Introduction of chains in plasmids of $E. coli$.
$(d)$ Extraction of chain $A$ and $B$ and combining them by creating disulphide bonds.
Choose the correct sequence of events.

In $1990$,which therapy was given to a four-year-old girl suffering from adenosine deaminase $(ADA)$ deficiency?

The first clinical gene therapy was given for the treatment of .........

Gene therapy was first used for the treatment of ..........

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