Level 1 - Absolute Beginner
Scientists are trying a new way to fix DNA inside a person. DNA is like a long set of instructions in every cell. Sometimes a small mistake in it causes a disease.
The new tool is called prime editing. It can change small parts of DNA. A company called Prime Medicine says it treated the first person in its trial this month.
The disease is called Wilson disease. In this disease, the body keeps too much copper. This can hurt the liver. The scientists hope the edit will fix the problem. Results are expected next year.
- DNA
- The instructions inside every cell.
- cell
- The smallest living part of a body.
- disease
- A sickness.
- liver
- An organ that cleans the blood.
- copper
- A reddish metal.
- trial
- A test of a new medicine.
- edit
- To change something.
- treat
- To give medical care.
Level 2 - Elementary
A report in the journal Nature on October 22 says doctors have started using prime editing to change DNA directly inside a human body. Prime editing is a very exact gene editing tool that works like a word processor for DNA.
The trial is for Wilson disease, a rare inherited illness. People with it cannot remove extra copper, and the metal builds up, especially in the liver. The treatment aims to rewrite DNA in liver cells to fix the fault.
Prime Medicine, a company in Cambridge, Massachusetts, said it treated the first person in its trial earlier this month. It expects to share the first results next year. An earlier prime editing treatment, for a teenager with chronic granulomatous disease, worked on cells in a different way.
- gene
- A part of DNA that carries one instruction.
- inherited
- Passed from parents to children.
- rare
- Not common.
- exact
- Very correct.
- builds up
- Slowly collects in one place.
- rewrite
- To write again.
- fault
- A mistake or problem.
- journal
- A science magazine for experts.
Level 3 - Intermediate
Nature reported on October 22 that prime editing, a precise form of gene editing, is being used to alter DNA directly inside a human body in a clinical trial for the first time. The target is Wilson disease, a rare inherited disorder in which the body cannot clear excess copper, which accumulates in the liver and brain.
Unlike earlier CRISPR tools that cut both strands of DNA, prime editing is designed to rewrite short stretches of the genetic code with fewer unintended changes. In the trial, the edit is aimed at liver cells, and Prime Medicine of Cambridge, Massachusetts, says it dosed its first participant earlier this month, with first results promised next year.
Researchers urge caution. A single patient says little about safety or lasting benefit, and earlier prime editing work, such as the treatment of a teenager with chronic granulomatous disease, involved cells handled differently. Even so, delivering the editor safely to enough liver cells would mark a major step toward one time treatments for genetic disorders.
- precise
- Exact and accurate.
- accumulate
- To collect gradually.
- disorder
- An illness that disrupts normal function.
- strand
- A single thread of a molecule.
- participant
- A person who takes part in a study.
- dose
- To give a measured amount of medicine.
- unintended
- Not planned.
- clinical trial
- A test of a treatment in people.
Level 4 - Advanced
Nature reported on October 22 that prime editing, the most surgical of the gene editing techniques, has entered a clinical trial that rewrites DNA directly within a living human, a threshold the field has approached for years. The disease in the crosshairs is Wilson disease, a rare inherited disorder in which defective copper handling lets the metal accumulate in the liver and brain.
Where first generation CRISPR severs both strands of the double helix and relies on the cell's repair crews, prime editing writes new sequence by copying from a template, which promises fewer stray alterations. Prime Medicine, based in Cambridge, Massachusetts, says it dosed the first participant earlier this month, targeting liver cells, and expects to disclose initial findings next year.
Specialists temper their enthusiasm. A single recipient reveals little about durability, immune reactions or off target effects, and earlier prime editing in a teenager with chronic granulomatous disease worked on cells handled outside the body. Yet if the editor can be delivered safely and efficiently to the liver, the approach could herald one time cures for conditions long managed only with lifelong drugs.
- threshold
- A point at which something new begins.
- surgical
- Extremely precise.
- defective
- Not working properly.
- sever
- To cut apart.
- template
- A pattern to copy from.
- temper
- To moderate.
- durability
- The ability to last.
- herald
- To announce or signal the coming of.
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