In gene therapy, which vector is commonly used to deliver functional genes into a patient's cells?
Bacteria
Plasmids
Modified viruses
Liposomes
Related Questions
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
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.
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.
In gene therapy, which vector is commonly used to deliver functional genes into a patient's cells?
Bacteria
Plasmids
Modified viruses
Liposomes
