Toolkit Items

Browse the toolkit beneath workflows. The mechanism branch runs mechanism -> architecture -> component, while the technique branch runs from high-level approaches down to concrete methods.

7 items matching 1 filter

Mechanism Branch

Layer 1

Mechanisms

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Top-level concepts: biophysical action modes such as heterodimerization, photocleavage, or RNA binding.

Layer 2

Architectures

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Arrangements that realize or deploy mechanisms, including switches, construct patterns, and delivery strategies.

Layer 3

Components

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Low-level parts and sequence-defined elements used inside architectures, including protein domains and RNA elements.

Technique Branch

Layer 1

Approaches

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High-level engineering practices such as computational design, directed evolution, sequence verification, and functional assay.

Layer 2

Methods

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Concrete methods used to design, build, verify, or characterize engineered systems.

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viral delivery

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Adeno-associated virus

Delivery Strategy
Since 2017transgene expressionTranslation Controlviral deliveryComputational DesignSequence Verification

Adeno-associated virus (AAV) is a viral delivery harness used to package and express CRISPR genome-editing components in vivo. In the cited literature, AAV supports single-vector delivery when smaller Cas9 orthologues and their chimeric guide RNAs fit within AAV packaging constraints, enabling robust in vivo genome editing.

CFBacMamMusHumTxRep
Ev 78Rep 63Pr 71

adeno-associated virus (AAV) particles

Delivery Strategy
Since 2018viral deliveryComputational Design

Adeno-associated virus (AAV) particles were used as a delivery harness for the BphP1-QPAS1-based TA optogenetic system in neurons. In the cited ChemBioChem study, this application was enabled by the small size of the QPAS1 component.

CFBacMamMusHumTxRep
Ev 28Rep 9Pr 71

recombinant adeno-associated virus (rAAV)

Delivery Strategy
Since 2015intracellular reconstitution via intein-mediated protein splicingviral delivery

Recombinant adeno-associated virus (rAAV) is a viral delivery harness used in the cited study to package and deliver an intein-mediated split-Cas9 system for gene therapy. In this context, rAAV enabled cellular delivery of Cas9 fragments that efficiently reconstituted nuclease activity after intein-mediated protein splicing.

CFBacMamMusHumTxRep
Ev 28Rep 9Pr 71

viral transduction

Delivery Strategy
Since 2020viral delivery

Viral transduction is a conventional delivery harness used to introduce CRISPR components for gene editing. In the cited review context, it is presented as an established delivery approach whose performance for CRISPR cargo can be limited.

CFBacMamMusHumTxRep
Ev 20Rep 9Pr 71

AAV8

Delivery Strategy
viral delivery

Furthermore, when delivered via AAV8, Cj4Cas9 achieves efficient genome editing of the Pcsk9 gene in mouse liver, leading to reduced serum cholesterol and LDL-C levels.

CFBacMamMusHumTxRep
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