A patient exhibits severe combined immunodeficiency (SCID) due to a deficiency in adenosine deaminase (ADA). Which gene therapy approach is MOST likely to provide a long-term solution?
In vivo gene therapy using a retroviral vector directly injected into the bone marrow.
Administering purified ADA enzyme intravenously.
Ex vivo gene therapy using autologous hematopoietic stem cells.
Bone marrow transplant from an HLA-matched sibling.
Related Questions
In the production of monoclonal antibodies (mAbs), hybridomas are created. What crucial property makes hybridomas suitable for large-scale mAb production?
Ability to phagocytose antigens.
Short lifespan, ensuring consistent antibody quality.
Continuous cell division and antibody secretion.
Capacity to produce diverse antibody isotypes.
Transgenic animals are used for:
Producing antibiotics
Testing the safety of vaccines
Diagnosing genetic diseases
Treating bacterial infections
A drug obtained through genetic engineering and useful for treating infertility is
Calcitonin
Chorionic gonadotropin
Interleukin
Tissue plasminogen activator
For the early diagnosis of a disease, which of the following is NOT typically used?
Amniocentesis
Ultrasound
Chorionic villus sampling (CVS)
Physical therapy
ELISA is a technique used to detect:
Specific DNA sequences
RNA transcripts
Antigens or antibodies
Gene mutations
Consider the following statements about therapeutic drugs
I. The recombinant DNA technology is used for production of therapeutic drugs which are safe and effective
II. It avoid unwanted immunological responses, commonly observed with similar products isolated from non-human sources
III. About thirty recombinant therapeutics have been approved for human use in the world including India
Which of the statements given above are correct?
I and II
I and III
II and III
I, II and III
A patient exhibits severe combined immunodeficiency (SCID) due to a deficiency in adenosine deaminase (ADA). Which gene therapy approach is MOST likely to provide a long-term solution?
In vivo gene therapy using a retroviral vector directly injected into the bone marrow.
Administering purified ADA enzyme intravenously.
Ex vivo gene therapy using autologous hematopoietic stem cells.
Bone marrow transplant from an HLA-matched sibling.
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
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.
Molecular diagnosis utilizes which biological molecule to detect diseases?
Carbohydrates
Lipids
Proteins
DNA/RNA
