Gene Therapy — Prelims Questions

Which of the following statements about Adeno-associated Viruses (AAVs) as gene therapy vectors is/are correct? 1. They are known for their high immunogenicity, limiting repeat dosing. 2. They can infect both dividing and non-dividing cells. 3. Zolgensma and Luxturna are examples of AAV-based gene therapies. Select the correct answer using the code given below:

Consider the following statements regarding gene therapy in India: 1. The New Drugs and Clinical Trials Rules, 2019, explicitly include 'gene therapeutic products' under the definition of 'new drug'. 2. Germline gene therapy is permitted in India under strict ethical guidelines for therapeutic purposes. 3. The ICMR's National Guidelines for Stem Cell Research and Therapy also cover gene therapy. Which of the statements given above is/are correct?

Which of the following diseases can be targeted by CAR-T cell therapy? 1. Spinal Muscular Atrophy (SMA) 2. Leber Congenital Amaurosis (LCA) 3. Certain types of Leukemias and Lymphomas 4. Sickle Cell Disease Select the correct answer using the code given below:

Consider the following statements regarding the ethical considerations of gene therapy: 1. Somatic gene therapy is generally considered ethically acceptable as its effects are not heritable. 2. Germline gene therapy is widely prohibited globally due to concerns about altering the human gene pool. 3. The high cost of gene therapies raises significant ethical questions about equitable access and affordability. Which of the statements given above are correct?

Which of the following is NOT a non-viral method for gene delivery in gene therapy?