Chapter 1
The bacterial origin story
Chapter 2
How the genetic scissors work
Chapter 3
Editing life to cure disease
Chapter 4
Rewriting the natural world
YOUR GOAL
Master of How CRISPR edits life
Ch 3 · Editing life to cure disease
diego CRISPR Gene Editing
Wake up the silent gene (wake up!) / Fetal blood runs healthy and clean (so clean!)
diego CRISPR can be injected directly into the eye to repair genetic mutations that cause inherited blindness.
diego Scientists are using CRISPR to locate and destroy viral DNA that hides inside human cells, including HIV.
Fun Fact
Fetal hemoglobin trick: The drug Casgevy cures sickle cell by turning back on a gene we only use as babies.
Glossary
crispr (noun)
A genetic engineering tool used to make precise, targeted changes to the DNA of a living organism. In 2019, clinicians first used this molecular tool to treat Victoria Gray's hereditary sickle cell disease.
A genetic engineering tool used to make precise, targeted changes to the DNA of a living organism. In 2019, clinicians first used this molecular tool to treat Victoria Gray's hereditary sickle cell disease.
Quiz
In December 2023, the US FDA approved Casgevy, the first CRISPR drug, to treat which genetic disorder?
Quiz
In 2024, researchers in Amsterdam successfully used CRISPR to eliminate which latent virus from infected cells?
Quiz
What are scientists developing to prevent off-target cuts by requiring a 100% perfect genetic match?
Quiz
Why is the human eye an ideal organ for testing direct, in-body CRISPR gene therapies?
Quiz
What protein's production is boosted in patient stem cells to cure sickle cell disease using Casgevy?
Quiz
What term is used when CRISPR accidentally cuts DNA at a site that is highly similar but not the intended target?
Quiz
What rare genetic blindness was targeted in the 2024 BRILLIANCE clinical trial using direct CRISPR injections?