Podcast
Questions and Answers
What is the primary gene missing or reduced in patients with ADA-SCID?
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?
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?
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?
Which approach has been increasingly used as a curative method for ADA-SCID once the diagnosis is established?
What is the primary issue associated with insertional mutagenesis in gene therapy?
What is the primary issue associated with insertional mutagenesis in gene therapy?
What is a key characteristic of the Umbilical Cord Blood stem cell transplantation approach?
What is a key characteristic of the Umbilical Cord Blood stem cell transplantation approach?
Which clinical study design was used for evaluating the Lentivirus vector treatment for ADA-SCID?
Which clinical study design was used for evaluating the Lentivirus vector treatment for ADA-SCID?
What is the main genetic error in patients with CYBB-associated SCID?
What is the main genetic error in patients with CYBB-associated SCID?
Which of the following is NOT a feature of ADA-SCID?
Which of the following is NOT a feature of ADA-SCID?
What is the primary aim of using lentivirus vectors in gene therapy for ADA-SCID?
What is the primary aim of using lentivirus vectors in gene therapy for ADA-SCID?
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