Geen biologische achtergrond nodig
Leer gene editing, vanaf nul
Een reeks korte gidsen: wat DNA is, wat een gen is, hoe CRISPR een sequentie vindt en verandert, en vervolgens de geavanceerde methoden die daarna kwamen. Lees ze op volgorde of spring naar een willekeurig onderdeel.
24 gidsen. Elke gids begint met een antwoord in begrijpelijke taal en gaat daarna dieper in op het onderwerp. Analogieën worden vrijelijk gebruikt — en elke analogie wordt vergezeld van een toelichting over precies waar ze ophouden te kloppen, want een half begrepen analogie is hoe de meeste misvattingen over dit vakgebied ontstaan.
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Start here
Het vocabulaire waar alles op voortbouwt.
Gids · 4 min lezen
What is DNA?
DNA is a long chemical chain that stores the instructions for building and running a living thing, written in an…
Gids · 4 min lezen
What is a gene?
A gene is a stretch of DNA that carries the instructions for making one particular thing the cell needs — usually a…
Gids · 3 min lezen
What is a chromosome?
A chromosome is one very long DNA molecule packaged with proteins; humans normally have 46, in 23 pairs.
Gids · 3 min lezen
What is a genome?
A genome is the complete set of genetic instructions in an organism — every chromosome, every gene, and everything in…
Gids · 3 min lezen
What is RNA?
RNA is DNA's working copy — a temporary, single-stranded version the cell makes when it needs to use a gene.
Gids · 5 min lezen
What is a mutation?
A mutation is a change in DNA sequence. Most do nothing, some cause disease, and a few are beneficial.
Gids · 5 min lezen
What is gene editing?
Gene editing is changing the DNA sequence already inside a living cell, at a chosen location.
Gids · 6 min lezen
What is CRISPR?
CRISPR is a bacterial immune system that scientists reprogrammed into a tool for finding and changing a chosen piece of…
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CRISPR basics
Hoe het systeem stap voor stap echt werkt.
Gids · 6 min lezen
How CRISPR works, step by step
A guide RNA finds the target, the Cas protein cuts, and the cell's repair machinery makes the actual change.
Gids · 4 min lezen
What Cas proteins do
Cas proteins are the machinery of the CRISPR system: they carry the guide, search the genome, and cut what matches.
Gids · 4 min lezen
What guide RNA does
The guide RNA is the address. It carries a short sequence matching the target and holds the Cas protein in place.
Gids · 5 min lezen
How cells repair DNA — and why it decides the edit
The cell, not the editor, makes the change. Which repair pathway it uses determines what you actually get.
Gids · 5 min lezen
What off-target editing means
An off-target edit is a change made somewhere other than the intended site, because the guide tolerated a mismatch.
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Intermediate
Wat editing met een gen kan doen, en hoe het in een lichaam wordt gebracht.
Gids · 5 min lezen
Knockouts, insertions and deletions
The three basic things editing does to a gene: switch it off, take something out, or put something in.
Gids · 6 min lezen
Delivery: the real bottleneck
Getting the editing machinery into the right cells, in enough of them, without harm — the problem that decides which…
Gids · 5 min lezen
Ex vivo and in vivo editing
Ex vivo means editing cells outside the body and putting them back; in vivo means editing them where they are.
Gids · 5 min lezen
Viral vectors
Modified viruses used as delivery vehicles — very good at getting inside cells, which is exactly what they evolved to…
Gids · 4 min lezen
Lipid nanoparticles
Tiny fat bubbles that carry genetic cargo into cells — the delivery technology that made in vivo CRISPR possible.
Gids · 4 min lezen
Turning genes up and down
Changing how loudly a gene is expressed, without changing a single letter of its sequence.
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Advanced
De methoden die na Cas9 kwamen, en waar het vakgebied naartoe gaat.
Gids · 4 min lezen
Multiplex editing
Making several edits in the same cell at once — necessary for cell therapy, and riskier than making one.
Gids · 4 min lezen
CRISPR diagnostics
Turning CRISPR's habit of chopping up nearby nucleic acid into a fast, cheap test for a specific sequence.
Gids · 6 min lezen
Why gene therapies cost millions
The economics of a one-time treatment for a small population — and why the arithmetic that produces a $2.2 million…
Gids · 5 min lezen
How a gene-editing company works
What a pre-revenue biotechnology company actually is, and which numbers describe it — information, not investment…
Gids · 6 min lezen
How to read a clinical trial result
What a Phase 1 result does and does not tell you, and the questions worth asking before you believe a headline.