Question

The first clinical gene therapy was given in 1990 to a four year old girl suffering from
Adenosine Deaminase Deficiency (ADA) . Could you suggest a possible cure for such
a disorder with the knowledge of gene therapy and its types?

TNSCERT Class 10 Biology Ch 1 Heredity and Evolution

Answer 1

The most common methods of gene therapy is direct incorporation of genes to cells that have defective gene copies. This perhaps might be the most direct method of curing a case of the Adenosine Deaminase Deficiency.
Repairing mutations :
this might perhaps a potent treatment as it involves the repair of the defective gene through mRNA incorporation to the chromosome 20 that will aid code functional adenosine deaminase.
Note: Since the ADA deficiency only involves mutations of genes and not protein dis-functions such as improper regulation,gain-of- function or dominant negative, the gene silencing form of gene therapy may not be the right choice of treatment.

Hope you find this helpful :)