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Lentivirus Vectors and ADA-SCID Syndrome
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Lentivirus Vectors and ADA-SCID Syndrome

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Questions and Answers

What is the primary gene missing or reduced in patients with ADA-SCID?

  • CYBA
  • ADA (correct)
  • CYBB
  • NCF1

    • Which of the following is a consequence of untreated immune deficiency in patients with ADA-SCID?

  • Variable platelet correction
  • Increased gene expression
  • Tertiary structure formation
  • Profound pan-lymphopenia (correct)

    • What is the primary initiating element for the transcription of the transgene in lentivirus vectors used in research?

  • WPRE/3’UTR
  • Sequence that enhances expression
  • Promoter (correct)
  • Transgene

    • Which approach has been increasingly used as a curative method for ADA-SCID once the diagnosis is established?

    <p>AlloHSCT</p> Signup and view all the answers

    What is the primary issue associated with insertional mutagenesis in gene therapy?

    <p>Uncontrolled gene expression activation</p> Signup and view all the answers

    What is a key characteristic of the Umbilical Cord Blood stem cell transplantation approach?

    <p>HLA-matched sibling donor requirement</p> Signup and view all the answers

    Which clinical study design was used for evaluating the Lentivirus vector treatment for ADA-SCID?

    <p>Prospective, non-randomised</p> Signup and view all the answers

    What is the main genetic error in patients with CYBB-associated SCID?

    <p>Mutations in CYBB</p> Signup and view all the answers

    Which of the following is NOT a feature of ADA-SCID?

    <p>Increased platelet count</p> Signup and view all the answers

    What is the primary aim of using lentivirus vectors in gene therapy for ADA-SCID?

    <p>Inserting functional ADA gene into patients' cells</p> Signup and view all the answers

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