A person is born with a hereditary disease with a weakened immune syst...
Gene therapy is the technique which can be used.Introduction of gene isolated from bone marrow cells at early embryonic stages(bone marrow transplantation) is a permanent cure.This is caused due to deficiency of ADA(Adenosine deaminase)
A person is born with a hereditary disease with a weakened immune syst...
The Hereditary Disease and its Deficiency
A person born with a hereditary disease characterized by a weakened immune system due to the deficiency of an enzyme faces significant health challenges. The deficiency of this specific enzyme impairs the body's ability to effectively fight off infections and diseases, making the individual more susceptible to various pathogens. To provide a complete cure for this disease, it is crucial to identify the deficient enzyme and devise a technique that can address the underlying cause.
Identifying the Deficient Enzyme
Determining the specific enzyme responsible for the disease is a critical step in developing an effective cure. This can be achieved through thorough genetic testing and analysis. By examining the individual's DNA, scientists can identify any mutations or alterations in the genes that code for the production of the enzyme. Comparing the results with a healthy individual's genetic profile can pinpoint the precise enzyme that is deficient in the affected person.
Technique for Complete Cure
To provide a comprehensive cure for this hereditary disease, a potential technique can be employed:
1. Gene Therapy:
Gene therapy holds immense promise in treating genetic disorders. In this technique, the deficient gene responsible for the enzyme deficiency is targeted and corrected. The process involves introducing a functional copy of the gene into the affected individual's cells. This can be achieved through various methods, such as viral vectors or gene editing tools like CRISPR-Cas9.
2. Viral Vectors:
Viral vectors can be utilized to deliver the corrected gene into the cells. These vectors are modified viruses that can efficiently transfer the desired genetic material into the patient's cells without causing harm. By using a specific viral vector, the corrected gene can be inserted into the affected cells, allowing them to produce the deficient enzyme.
3. CRISPR-Cas9:
CRISPR-Cas9 is a revolutionary gene editing tool that enables precise modifications to the DNA. It can be employed to directly edit the genes responsible for the deficient enzyme. By using CRISPR-Cas9, scientists can remove or repair the mutated gene, allowing the cells to produce the functional enzyme.
4. Stem Cell Therapy:
Stem cell therapy can also be considered as a potential technique for the complete cure of this hereditary disease. By utilizing stem cells, which have the ability to differentiate into various cell types, scientists can generate healthy cells capable of producing the deficient enzyme. These healthy cells can then be transplanted into the affected individual, replacing the diseased cells and restoring proper immune function.
Conclusion
In conclusion, a hereditary disease characterized by a weakened immune system due to the deficiency of a specific enzyme can potentially be completely cured through techniques such as gene therapy, viral vectors, CRISPR-Cas9, or stem cell therapy. Identifying the deficient enzyme and developing a targeted approach to address the underlying cause of the disease is crucial for providing an effective and lasting cure. These innovative techniques offer hope for individuals affected by this hereditary disease, ultimately improving their quality of life and overall health.