Insertional mutagenesis, a potential risk of gene therapy, refers to:
Immune response against the therapeutic gene
Degradation of the therapeutic gene
Unintended insertion of the therapeutic gene disrupting other genes
Failure of the therapeutic gene to be expressed
Related Questions
In the context of genetic modification of human lymphocytes, which vector is known for its ability to integrate its genetic material into the host genome?
Retrovirus
Plasmid
Cosmid
Yeast Artificial Chromosome (YAC)
A plasmid containing complete gene copied from human genome is introduced in a bacterial cell, but the bacteria is unable to make a working copy of the protein because
It cannot recognize the start codon in mRNA
Of the absence of similar amino acids in bacteria
Of the absence of similar splicing machinery
The foreign gene cannot replicate in bacteria
Which of the following is the primary reason why gene therapy for ADA deficiency requires repeated lymphocyte infusions?
Limited lifespan of the modified lymphocytes.
Immune rejection of the modified lymphocytes.
Loss of ADA gene expression over time.
Inability of the modified lymphocytes to produce functional ADA.
Which of the following is NOT a crucial consideration when designing a CRISPR-Cas9 system for gene therapy targeting a specific genetic disorder?
Ensuring precise targeting of the Cas9 enzyme to the mutated gene
Minimizing off-target effects of the Cas9 enzyme
Choosing an appropriate delivery system for the CRISPR-Cas9 components
Enhancing the host's immune response to the Cas9 enzyme
A plasmid containing complete gene copied from human genome is introduced in a bacterial cell, but the bacteria is unable to make a working copy of the protein because
It cannot recognize the start codon in mRNA
Of the absence of similar amino acids in bacteria
Of the absence of similar splicing machinery
The foreign gene cannot replicate in bacteria
One of the challenges of gene therapy is ensuring the therapeutic gene reaches the target cells. This process is known as:
Gene expression
Gene regulation
Gene delivery
Gene silencing
Gene therapy can involve modifying genes inside the body (in vivo) or outside the body (ex vivo). Which method involves directly introducing the therapeutic gene into the patient's cells within their body?
In vivo
Ex vivo
Both in vivo and ex vivo
Neither in vivo nor ex vivo
The site of the production of ADA in the body is
Bone marrow
Lymphocytes
Blood plasma
Monocytes
What is the role of a 'functional gene' in gene therapy?
To cause the disease
To protect against infections
To replace a faulty gene
To enhance immune response
SCID is a genetic disorder affecting which body system, making it a target for gene therapy?
Nervous system
Digestive system
Immune system
Endocrine system
