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Question

Which of the following statements about gene therapy are CORRECT?
[P] Affected individuals, but not their progeny, can be cured through germline gene therapy
[Q] Affected individuals, as well as their progeny, can be cured through germline gene therapy
[R] Affected individuals, but not their progeny, can be cured through somatic gene therapy
[S] Affected individuals, as well as their progeny, can be cured through somatic gene therapy

The correct answer is
Q and R only

Gene Therapy: Germline vs. Somatic Statements

This explanation analyzes the key differences between germline and somatic gene therapy to determine the correctness of the provided statements.

Somatic Gene Therapy Explained

  • Focus: Targets the somatic cells (body cells) of an individual.
  • Effect: Modifications affect only the treated individual. These changes are not passed down to offspring.
  • Statement Analysis: Consequently, somatic gene therapy can cure affected individuals, but their progeny will not be affected by the therapy. This makes statement [R] correct and statement [S] incorrect.

Germline Gene Therapy Explained

  • Focus: Targets the germ cells (sperm or egg cells) or the cells of an early embryo.
  • Effect: Modifications are incorporated into the genetic makeup and are heritable, meaning they can be passed on to future generations (progeny).
  • Statement Analysis: Therefore, germline gene therapy has the potential to cure affected individuals and also prevent the condition in their progeny. This makes statement [Q] correct and statement [P] incorrect.

Conclusion on Statements

Based on the distinct characteristics of each type of gene therapy:

  • Statement [Q] accurately describes germline gene therapy's impact on individuals and their progeny.
  • Statement [R] accurately describes somatic gene therapy's impact on individuals without affecting progeny.

Statements [Q] and [R] are the correct descriptions.

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Important Questions from Genetic Disorders

  1. Match the genetic disorder (Column I) with its molecular basis (Column II)

    Column IColumn II
    P. Sickle-cell anemia1. Mutation in nucleotide excision repair
    Q. Xeroderma pigmentosum2. Trisomy of chromosome 21
    R. Tay-Sachs disease3. Mutation in $ \beta$-globin gene
    S. Down Syndrome4. Mutation in hexosaminidase A gene
  2. A pedigree of an inheritable disease is shown below.


    This inheritable disease is

  3. Match the genetic disorders in Column I to the corresponding underlying cause in Column II
    Column IColumn II
    P. Klinefelter Syndrome1. X-chromosome insufficiency
    Q. Turner syndrome2. DNA helicase mutations
    R. Bloom syndrome3. Nucleotide excision repair defects
    S. Xeroderma pigmentosum4. Extra X chromosome(s) in a male
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