A severely ill patient suffering from severe combined immunodeficiency (SCID)-X1 requires immediate lifesaving treatment. A compatible bone marrow donor is not available. Devise a treatment procedure that could potentially cure the disease without additional life- threatening risks to the patient

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A severely ill patient suffering from severe combined immunodeficiency (SCID)-X1 requires immediate lifesaving treatment. A compatible bone marrow donor is not available. Devise a treatment procedure that could potentially cure the disease without additional life- threatening risks to the patient

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question

Gene Therapy for SCID-X1

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layout

Aspect
Details

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aspects

Disease Cause
Why Not Bone Marrow
Gene Therapy Steps

            Preconditioning
            Expected Outcome
            Risks
            Advantages
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Disease Cause

Mutation in IL2RG gene (X-linked)

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Why Not Bone Marrow

No compatible donor available

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Gene Therapy Steps

  1. Isolate patient HSCs
    2. Insert correct IL2RG using lentiviral vector
    3. Re-infuse cells
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Preconditioning

Mild chemotherapy to aid engraftment

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Expected Outcome

Restoration of T, B, and NK cells → functional immune system

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Risks

Insertional mutagenesis (minimized with new vectors)

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Advantages

No GvHD, no donor needed, potentially curati