The genetic defect-Adenosine deaminase (ADA) deficiency may be cured permanently by

Options

A. Administering adenosine deaminase through Injection
B. Bone marrow transplantation
C. Enzyme replacement therapy.
D. Introducing isolated gene from marrow cells producing ADA into the cells at early embryonic stages.

Answer

To solve the question regarding the permanent cure for the genetic defect known as Adenosine deaminase (ADA) deficiency, we can follow these steps:

### Step 1: Understand ADA Deficiency
Adenosine deaminase (ADA) deficiency is a genetic disorder that affects the immune system. It is caused by mutations in the ADA gene, leading to a lack of the enzyme adenosine deaminase, which is crucial for the breakdown of adenosine. This deficiency results in the accumulation of toxic metabolites that damage the immune system.

**Hint:** Research the role of adenosine deaminase in the immune system and the consequences of its deficiency.

### Step 2: Identify Treatment Options
The available treatment options for ADA deficiency include:
1. Enzyme replacement therapy (ERT)
2. Bone marrow transplantation (BMT)
3. Gene therapy (introducing a functional ADA gene into the patient's cells)

**Hint:** List the pros and cons of each treatment option to understand their effectiveness.

### Step 3: Evaluate Treatment Efficacy
- **Enzyme Replacement Therapy (ERT):** This method involves administering the ADA enzyme through injections. While it can alleviate symptoms, it does not provide a permanent solution.
- **Bone Marrow Transplantation (BMT):** This can restore ADA production but is not always successful and depends on finding a compatible donor.
- **Gene Therapy:** This approach involves introducing a functional ADA gene into the patient's cells, potentially providing a permanent cure.

**Hint:** Consider which treatment option addresses the root cause of the deficiency rather than just managing symptoms.

### Step 4: Determine the Permanent Cure
Among the treatment options, the introduction of an isolated gene from bone marrow cells that produces ADA at early embryonic stages is the most promising for a permanent cure. This method aims to correct the genetic defect at its source.

**Hint:** Think about how gene therapy differs from other treatments in terms of addressing genetic disorders.

### Conclusion
The genetic defect Adenosine deaminase (ADA) deficiency may be cured permanently by introducing an isolated gene from marrow cells that produces ADA into the cells at early embryonic stages.

**Final Answer:** The correct answer is D: Reducing isolated gene from marrow cells at early embryonic stages.

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