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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?

AlloHSCT Signup and view all the answers

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

Uncontrolled gene expression activation Signup and view all the answers

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

HLA-matched sibling donor requirement Signup and view all the answers

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

Prospective, non-randomised Signup and view all the answers

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

Mutations in CYBB Signup and view all the answers

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

Increased platelet count Signup and view all the answers

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

Inserting functional ADA gene into patients' cells Signup and view all the answers