Principles and Mechanisms of Human Gene Therapy

A collection of question and answer flashcards covering key concepts from the lecture on human gene therapy.

What are the main types of gene therapy discussed in the lecture?

Gene replacement therapy, CAR-T therapy, RNA vaccines, Antisense therapy, RNAi therapy, Gene Editing.

What does the sense strand of DNA correspond to?

The coding strand that carries the information for protein synthesis.

What cellular event occurs during transcription?

The conversion of DNA into pre-mRNA by RNA polymerase II.

What is the first gene therapy approved for use, and what does it target?

Gendicine, which targets head and neck squamous cell carcinoma.

What is the mechanism of action for RNA vaccines?

They deliver mRNA that encodes a protein, triggering the immune response to produce antibodies.

What are some challenges associated with gene therapy?

DNA degradation by nucleases, need for intact delivery to the nucleus, avoiding immune responses, ensuring proper protein folding.

What does CAR-T therapy involve?

Genetically modifying T-cells to target and destroy cancer cells.

What is RNA interference (RNAi)?

A biological process through which RNA molecules inhibit gene expression by degrading mRNA.

What are the ethical concerns regarding germ line gene therapy?

The potential implications on genetic diversity and the ability to pass modified genes to future generations.

How does antisense therapy work?

It uses oligonucleotides to bind to mRNA, blocking its translation and potentially leading to its degradation.

What is p53, and why is it significant in cancer therapy?

A protein that checks for cellular stress and DNA damage; its reactivation could lead to tumor regression.

What are the proposed solutions to the challenges of gene therapy?

Use of biocompatible vectors, improved targeting methods, and ensuring appropriate expression levels.