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Which of the following could be a preman...

Which of the following could be a premanent cure for treatment of severe combined immunodeficiency (SCID)?

A

Bone marrow transplantation

B

Enzyme replacment therapy

C

Both (1) and (2)

D

Gene therapy

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AI Generated Solution

The correct Answer is:
### Step-by-Step Solution: 1. **Understanding SCID**: Severe Combined Immunodeficiency (SCID) is a genetic disorder characterized by a deficiency in both T and B lymphocyte systems, leading to a severely compromised immune system. 2. **Identifying the Cause**: The primary cause of SCID is a mutation in the ADA gene (adenosine deaminase gene), which is crucial for the immune system's function. 3. **Mechanism of the ADA Gene**: The ADA gene encodes the enzyme adenosine deaminase. A mutation in this gene results in a lack of functional enzyme, which is essential for the breakdown of toxic metabolites that can accumulate and harm lymphocytes. 4. **Gene Therapy as a Treatment**: Gene therapy involves introducing a normal, functional copy of the ADA gene into the patient's cells. This is typically done using a retrovirus that carries the healthy gene. 5. **Procedure**: The retrovirus containing the functional ADA gene is injected into the patient's bone marrow cells (ex vivo). These cells are then modified to incorporate the healthy gene. 6. **Integration into Host Cells**: The introduced gene can either integrate into the host's chromosomes at various sites or replace the defective gene, allowing for the production of the functional enzyme. 7. **Permanent Cure**: Unlike other treatments such as bone marrow transplantation or enzyme replacement therapy, which are temporary and require ongoing treatment, gene therapy aims to provide a permanent solution by correcting the underlying genetic defect. 8. **Conclusion**: Therefore, gene therapy is considered a permanent cure for SCID, as it addresses the root cause of the disorder by correcting the genetic mutation. ### Final Answer: The permanent cure for the treatment of severe combined immunodeficiency (SCID) is **gene therapy**. ---
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