Der umfassende Leitfaden zum Genome Editing.
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Technology · Nuclease editing

Cas14 and miniature Cas systems

Unusually small CRISPR nucleases — roughly a third the size of Cas9 — discovered in archaea and bacteriophages.

Discovery DNAsmallresearch
Laboratory research Demonstrated in the laboratory. There is no treatment in or near the clinic for this use.

Einfache Erklärung

The biggest practical problem in gene editing is not the editing, it is getting the machinery into the right cells. The most common delivery vehicle, a modified virus, has a strict luggage limit, and Cas9 barely fits. Cas14 and its relatives are miniature versions of the same kind of enzyme — small enough to leave room for everything else you need to pack.

Tiefer eintauchen

Cas14 proteins (reclassified within Cas12f) are compact type V effectors of roughly 400–700 amino acids, discovered in uncultivated archaea. CasΦ (Cas12j), found in huge bacteriophages, is similarly small and notable for recognising a minimal T-rich PAM and processing its own guide RNA. Their size makes them attractive for AAV delivery, where the ~4.7 kb packaging limit is a hard constraint; native activity in human cells is generally lower than SpCas9 and much of the work is engineering to improve it.

Why size is the point

Adeno-associated virus, the workhorse of in vivo gene delivery, cannot carry more than about 4.7 kilobases. SpCas9 alone consumes most of that budget before you add a promoter, a guide and any regulatory sequence. A nuclease a third the size changes what is possible to deliver in a single vector — which is why an enzyme with modest laboratory activity still attracts serious engineering effort.

Where it stands

These are research-stage tools. They are used in laboratories and are being engineered for higher activity, and no therapeutic programme based on them has reached the clinic.

Where it stands
A miniature Cas effector beside a conventional one, at the same scale. Illustration generated for The CRISPR Atlas — a visual aid, not a photograph or a literal depiction of molecular structure.

Sources

Connected in the Atlas

Every entry on this site is linked to the others it relates to. These connections are part of the record, not a search result.

Scientists

Jennifer Doudna
Nur zu Bildungszwecken Diese Seite ist eine Referenz, keine medizinische Beratung. Forschungs- und Zulassungsstatus können sich ändern; beachten Sie das oben angegebene Aktualisierungsdatum und gleichen Sie wichtige Informationen mit den aufgeführten Primärquellen ab.