Module 8: Human Genome Editing

Somatic (non-heritable)

occur in body cells other than reproductive cells and are not passed on to offspring.

Germline (heritable)

occur in the reproductive cells and can be passed on to offspring, making them heritable.

CRISPR-Cas9

A tool with “considerable potential” to edit the genome with ease.

2018 germline editing of twins to prevent HIV

2018, birth to twin girls who had been edited while embryos

safety, efficacy, consensus, necessity

-the relevant safety and efficacy issues have been resolved, based on appropriate understanding and balancing of risks, potential benefits, and alternatives,

-there is broad societal consensus about the appropriateness of the proposed application

disability rights critiques

Disability rights advocates caution that editing these traits may send a message that lives with such conditions are less valuable

Victoria Gray’s sickle cell trial

Victoria Gray, 34, of Forest, Miss., volunteered for one of the most anticipated medical experiments in decades: the first attempt to use the gene-editing technique CRISPR to treat a genetic disorder in the U.S

Off-target effects

unexpected variant that matches guide sequence

Prenatal genome editing

-small fetus, small dose

-Target many progenitor or stem cells at once helping multiple organs

-fetus is immunologically immature so CRISPR components can be delivered without an immune response

-Treatment before symptoms show up and irreversable damage

Precautionary Principle

is the ethical idea that we should avoid actions that might cause serious or irreversible harm, especially when we don’t fully understand the risks.

Imperative to Treat

is the ethical idea that we have a duty to act when we have the tools or knowledge to help someone who is suffering or at risk.