Gene Therapy and Genome Editing

A comprehensive set of flashcards summarizing key concepts, therapies, and strategies related to gene therapy and genome editing as covered in the lecture notes.

What is the CRISPR/Cas9 system known for?

A gene-editing technology that allows precise changes to an organism's DNA.

Who were the two women awarded the Nobel Prize for their discoveries in the field of DNA manipulation involving CRISPR/Cas9?

Emmanuelle Charpentier and Jennifer Doudna.

What is Fanzor in the context of gene editing?

An eukaryotic programmable RNA-guided endonuclease capable of cleaving DNA at specific target sites.

What does NICER stand for in gene editing?

Nicking Induced by CRISPR/Cas9 to Enhance Homologous Recombination.

What is the primary purpose of gene therapy in the treatment of genetic diseases?

To provide precise strategies for gene correction and to restore normal gene function.

What are non-viral vectors used for in gene editing?

Vehicles required to deliver genes or therapeutic material inside the cells, characterized by being non-toxic and highly efficient.

What distinguishes viral vectors from non-viral vectors in gene therapy?

Viral vectors are the most effective for gene transfer and can easily infect numerous cell types, while non-viral vectors are generally less efficient.

What is ADA-SCID, and why is it significant in gene therapy?

A rare, inherited disorder treated by the first-ever gene therapy clinical trial in humans, focusing on correcting ADA deficiency.

What does CAR T-Cell Immunotherapy involve?

Genetically engineering a patient's T cells to find and kill cancer cells.

What was the outcome of the 2016 trial using CRISPR for non-small cell lung cancer?

The trial concluded that CRISPR-Cas9 gene-edited T cells showed general safety and feasibility.

What is the significance of the LCA10 gene therapy trial?

It was the first-ever in vivo CRISPR-Cas9 genome editing medicine targeting a rare inherited eye disease.

How does checkpoint inhibition enhance T cell activity in cancer therapy?

By blocking inhibitory signals, checkpoint inhibitors enhance the activity of existing T cells to better attack cancer cells.

What is a key challenge in gene therapy related to delivery vectors?

Some therapeutic genes are too large to transfer using available delivery vectors.