Understanding Gene Therapy and the Human Genome

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What are introns and exons in the context of genes?

Introns are non-coding regions, while exons are the portion of DNA coded to proteins

Where is DNA commonly found?

Nucleus of every cell

What is the human genome?

A complete set of genes in a cell or organism

What causes single-gene disorders?

Mutations in specific genes

What are genetic disorders caused by?

Abnormalities in the DNA sequence

What does an X-linked disorder originate from?

Genes of sex chromosomes

What is the main cause of multifactorial inheritance?

Combination of genetic changes

Which type of vectors are modified viruses used to deliver DNA into target cells?

Adenovirus

What type of gene therapy introduces corrective genes to sperm cells, egg cells, or zygotes?

Reproductive gene therapy

What is the main drawback of using viral vectors in gene therapy?

Recovery of ability to cause disease

Which of the following is NOT a potential benefit of gene therapy?

Modifying organism capabilities

What is the primary focus of somatic gene therapy?

Introducing genes to target organ cells

8

Herpes simplex virus

What could be a potential outcome if gene therapy damages the gene pool?

Multigene disorder resulting from damaged gene pool

Which part of a cell or organism constitutes a complete set of genes?

Chromosomes

Which type of gene therapy eliminates abnormalities that could be transmitted to offspring?

Reproductive Gene Therapy

What is the term for diseases caused by abnormalities in the DNA sequence of an individual?

Genetic disorders

Where is DNA commonly found in a cell?

Nucleus

Which type of disorder is caused by mutations in specific genes?

Single-gene disorders

What are the non-coding regions of the DNA sequence called?

Introns

What is the main purpose of gene therapy?

To replace, repair, or regulate genes to treat or prevent diseases

Which type of gene therapy is not transmitted to offspring?

Somatic gene therapy

What could be a consequence of modifying an organism's genetic capabilities through gene therapy?

Alteration of its capabilities

What is a potential risk associated with using viral vectors in gene therapy?

Recovery of disease-causing ability

Which of the following is a disadvantage of gene therapy?

Ethical concerns

Study Notes

Gene Structure and Function

  • In the context of genes, introns are non-coding regions of DNA, while exons are coding regions that are expressed to form proteins.
  • DNA is commonly found in the nucleus of eukaryotic cells and in the cytoplasm of prokaryotic cells.

Human Genome and Genetic Disorders

  • The human genome refers to the complete set of genes in an individual.
  • Single-gene disorders are caused by mutations in a single gene, leading to a deficiency or malfunction of a specific protein.
  • Genetic disorders can be caused by mutations in specific genes, chromosomal abnormalities, or multifactorial inheritance.
  • X-linked disorders originate from mutations in genes located on the X chromosome.
  • Multifactorial inheritance is primarily caused by the interaction of multiple genetic and environmental factors.

Gene Therapy

  • Modified viruses are used as vectors to deliver DNA into target cells in gene therapy.
  • Germline gene therapy introduces corrective genes to sperm cells, egg cells, or zygotes, potentially eliminating abnormalities that could be transmitted to offspring.
  • The main drawback of using viral vectors in gene therapy is the potential for an immune response against the virus.
  • A potential benefit of gene therapy is the permanent correction of genetic disorders.
  • Somatic gene therapy focuses on introducing corrective genes to non-reproductive cells to treat genetic disorders.

Risks and Consequences of Gene Therapy

  • Damaging the gene pool could be a potential outcome if gene therapy alters the germ line, leading to unintended consequences for future generations.
  • A consequence of modifying an organism's genetic capabilities through gene therapy could be unforeseen effects on the organism's biology or ecosystem.
  • A potential risk associated with using viral vectors in gene therapy is the possibility of insertional mutagenesis, where the virus inserts into the host genome and causes unintended effects.
  • A disadvantage of gene therapy is the potential for limited accessibility and high cost, making it inaccessible to many individuals.

This outline provides an overview of genes, the human genome, genetic disorders, and gene therapy. It delves into the structure of chromosomes and DNA, and their role in inheritance and genetic disorders.

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