Chapter 8, Problem 8CT

Summary Introduction

To describe:

A way in which recombinant $\text{DNA}$ technology might be used to prevent translation of the protein in congenital disease caused due to synthesis of a mutated gene.

Introduction:

Mutated gene is caused due to substitution of a normal nucleotide with a different nucleotide leading to a change in function and specificity of the protein.

Gene therapy involves the recombinant DNA procedure, which seeks to cure genetic diseases by inserting, deleting, or altering the genes in a patient’s cells. Gene therapies include “fixing” a defective allele, inactivating an allele that increase disease susceptibility, or adding a functional allele to substitute for a defective one. Almost all gene therapies are still in the experimental stage or in the clinical trials.