A scientist is attempting to develop a novel gene therapy for cystic fibrosis. They are utilizing a modified adeno-associated virus (AAV) vector to deliver the functional CFTR gene. However, pre-clinical trials show limited efficacy due to pre-existing neutralizing antibodies against the AAV capsid in a significant portion of the target population. Which of the following strategies would be MOST effective in overcoming this limitation?
Increasing the dosage of the AAV vector.
Administering immunosuppressants prior to gene therapy.
Engineering AAV capsids with altered surface epitopes to evade pre-existing immunity.
Switching to a lentiviral vector delivery system.
Related Questions
RNA interference (RNAi) technology is used for therapeutic purposes by:
Introducing new genes into cells
Amplifying specific DNA sequences
Silencing specific genes involved in disease progression
Producing monoclonal antibodies
Which of the following cells cannot be grown under tissue which of culture condition?
Hela cells
Leucocytes
Kidney cells
Nerve cells
What is the role of gene therapy in medicine?
Producing genetically modified crops
Creating new antibiotics
Introducing genes into a patient's cells to treat disease
Diagnosing genetic disorders
Identify the technique that is NOT directly involved in the early diagnosis of diseases.
X-ray
CT scan
Blood culture
Surgery
A scientist is attempting to develop a novel gene therapy for cystic fibrosis. They are utilizing a modified adeno-associated virus (AAV) vector to deliver the functional CFTR gene. However, pre-clinical trials show limited efficacy due to pre-existing neutralizing antibodies against the AAV capsid in a significant portion of the target population. Which of the following strategies would be MOST effective in overcoming this limitation?
Increasing the dosage of the AAV vector.
Administering immunosuppressants prior to gene therapy.
Engineering AAV capsids with altered surface epitopes to evade pre-existing immunity.
Switching to a lentiviral vector delivery system.
Which technique is used to amplify a specific DNA sequence?
ELISA
PCR
Electrophoresis
Southern blotting
Which of the following techniques is NOT typically used in pharmacogenomics to identify genetic variations influencing drug response?
Single nucleotide polymorphism (SNP) analysis
Genome-wide association studies (GWAS)
Gene expression profiling
Satellite DNA profiling
For the early diagnosis of a disease, which of the following is NOT typically used?
Amniocentesis
Ultrasound
Chorionic villus sampling (CVS)
Physical therapy
Transgenic animals are used for:
Producing antibiotics
Testing the safety of vaccines
Diagnosing genetic diseases
Treating bacterial infections
The development of biosimilars offers a cost-effective alternative to innovator biologics. Which of the following is NOT a key characteristic that biosimilars must demonstrate compared to their reference product?
Highly similar physicochemical properties
Comparable efficacy and safety profile
Identical amino acid sequence
Similar pharmacokinetics and pharmacodynamics
