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Gene Editing lernen – von null an
Eine Reihe kurzer Guides: was DNA ist, was ein Gen ist, wie CRISPR eine Sequenz findet und verändert – und dann die fortgeschrittenen Methoden, die danach kamen. Einfach der Reihe nach lesen oder direkt einsteigen.
24 Leitfäden. Jeder beginnt mit einer leicht verständlichen Antwort und geht dann in die Tiefe. Analogien werden großzügig eingesetzt – und jede Analogie enthält einen Hinweis darauf, wo genau sie aufhört zu stimmen, denn ein halb verstandenes Bild ist der Ursprung der meisten Missverständnisse in diesem Feld.
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Start here
Das Vokabular, auf dem alles andere aufbaut.
Leitfaden · 4 Min. Lesezeit
What is DNA?
DNA is a long chemical chain that stores the instructions for building and running a living thing, written in an…
Leitfaden · 4 Min. Lesezeit
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…
Leitfaden · 3 Min. Lesezeit
What is a chromosome?
A chromosome is one very long DNA molecule packaged with proteins; humans normally have 46, in 23 pairs.
Leitfaden · 3 Min. Lesezeit
What is a genome?
A genome is the complete set of genetic instructions in an organism — every chromosome, every gene, and everything in…
Leitfaden · 3 Min. Lesezeit
What is RNA?
RNA is DNA's working copy — a temporary, single-stranded version the cell makes when it needs to use a gene.
Leitfaden · 5 Min. Lesezeit
What is a mutation?
A mutation is a change in DNA sequence. Most do nothing, some cause disease, and a few are beneficial.
Leitfaden · 5 Min. Lesezeit
What is gene editing?
Gene editing is changing the DNA sequence already inside a living cell, at a chosen location.
Leitfaden · 6 Min. Lesezeit
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
Wie das System wirklich funktioniert – Schritt für Schritt.
Leitfaden · 6 Min. Lesezeit
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.
Leitfaden · 4 Min. Lesezeit
What Cas proteins do
Cas proteins are the machinery of the CRISPR system: they carry the guide, search the genome, and cut what matches.
Leitfaden · 4 Min. Lesezeit
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.
Leitfaden · 5 Min. Lesezeit
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.
Leitfaden · 5 Min. Lesezeit
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
Was Editing mit einem Gen machen kann und wie es in einen Körper eingebracht wird.
Leitfaden · 5 Min. Lesezeit
Knockouts, insertions and deletions
The three basic things editing does to a gene: switch it off, take something out, or put something in.
Leitfaden · 6 Min. Lesezeit
Delivery: the real bottleneck
Getting the editing machinery into the right cells, in enough of them, without harm — the problem that decides which…
Leitfaden · 5 Min. Lesezeit
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.
Leitfaden · 5 Min. Lesezeit
Viral vectors
Modified viruses used as delivery vehicles — very good at getting inside cells, which is exactly what they evolved to…
Leitfaden · 4 Min. Lesezeit
Lipid nanoparticles
Tiny fat bubbles that carry genetic cargo into cells — the delivery technology that made in vivo CRISPR possible.
Leitfaden · 4 Min. Lesezeit
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
Die Methoden, die nach Cas9 kamen, und wohin sich das Fachgebiet entwickelt.
Leitfaden · 4 Min. Lesezeit
Multiplex editing
Making several edits in the same cell at once — necessary for cell therapy, and riskier than making one.
Leitfaden · 4 Min. Lesezeit
CRISPR diagnostics
Turning CRISPR's habit of chopping up nearby nucleic acid into a fast, cheap test for a specific sequence.
Leitfaden · 6 Min. Lesezeit
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…
Leitfaden · 5 Min. Lesezeit
How a gene-editing company works
What a pre-revenue biotechnology company actually is, and which numbers describe it — information, not investment…
Leitfaden · 6 Min. Lesezeit
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.