Toolkit/adeno-associated virus delivery

adeno-associated virus delivery

Delivery Strategy·Research·Since 2020

Also known as: AAV delivery

Taxonomy: Mechanism Branch / Architecture. Workflows sit above the mechanism and technique branches rather than replacing them.

Summary

Adeno-associated virus delivery is a viral gene delivery harness used to deploy the far-red light-induced split-Cre recombinase (FISC) system in vivo. In the cited study, AAV delivery enabled implementation of optogenetically controlled genome engineering in living systems.

Usefulness & Problems

Why this is useful

This delivery harness is useful because it provides an in vivo route for introducing the FISC optogenetic recombination system. In the cited mouse study, this supported spatiotemporally controlled, non-invasive genome engineering when combined with far-red light activation.

Problem solved

AAV delivery addresses the practical problem of getting the FISC genetic components into living tissues for in vivo operation. The supplied evidence supports successful deployment of FISC by AAV, but does not further specify packaging strategy, tropism, or dosing details.

Published Workflows

Objective: Develop a compact and controllable dCas12f-based CRISPRa platform suitable for programmable in vivo endogenous gene activation and therapeutic application.

Why it works: The abstract states that HEAL was engineered to enhance DNA binding, nuclear localization, and transactivator recruitment, and that compact dCas12f architecture addresses AAV size constraints while inducible variants add remote and precise control.

enhanced DNA bindingenhanced nuclear localizationtransactivator recruitment through MS2 coat protein binding to sgRNA-embedded MS2 aptamersprotein and guide architecture engineeringAAV deliveryinducible control design

Taxonomy & Function

Primary hierarchy

Mechanism Branch

Architecture: A delivery strategy grouped with the mechanism branch because it determines how a system is instantiated and deployed in context.

Techniques

No technique tags yet.

Target processes

No target processes tagged yet.

Implementation Constraints

The available evidence indicates use of adeno-associated virus to deliver the FISC system in vivo, in a mouse context from the cited study. No additional practical details are provided in the supplied text regarding serotype selection, promoter design, genome configuration, production method, or administration route.

The evidence only states that AAV delivery was used successfully for FISC deployment and does not report vector serotype, cargo architecture, expression levels, or quantitative delivery performance. Independent replication and generalization beyond this single reported application are not established by the supplied evidence.

Validation

Cell-freeBacteriaMammalianMouseHumanTherapeuticIndep. Replication

Supporting Sources

Ranked Claims

Claim 1application scopesupports2020Source 1needs review

The FISC system expands the optogenetic toolbox for DNA recombination to enable spatiotemporally controlled, non-invasive genome engineering in living systems.

Thus, the FISC system expands the optogenetic toolbox for DNA recombination to achieve spatiotemporally controlled, non-invasive genome engineering in living systems.
Claim 2application scopesupports2020Source 1needs review

The FISC system expands the optogenetic toolbox for DNA recombination to enable spatiotemporally controlled, non-invasive genome engineering in living systems.

Thus, the FISC system expands the optogenetic toolbox for DNA recombination to achieve spatiotemporally controlled, non-invasive genome engineering in living systems.
Claim 3application scopesupports2020Source 1needs review

The FISC system expands the optogenetic toolbox for DNA recombination to enable spatiotemporally controlled, non-invasive genome engineering in living systems.

Thus, the FISC system expands the optogenetic toolbox for DNA recombination to achieve spatiotemporally controlled, non-invasive genome engineering in living systems.
Claim 4application scopesupports2020Source 1needs review

The FISC system expands the optogenetic toolbox for DNA recombination to enable spatiotemporally controlled, non-invasive genome engineering in living systems.

Thus, the FISC system expands the optogenetic toolbox for DNA recombination to achieve spatiotemporally controlled, non-invasive genome engineering in living systems.
Claim 5application scopesupports2020Source 1needs review

The FISC system expands the optogenetic toolbox for DNA recombination to enable spatiotemporally controlled, non-invasive genome engineering in living systems.

Thus, the FISC system expands the optogenetic toolbox for DNA recombination to achieve spatiotemporally controlled, non-invasive genome engineering in living systems.
Claim 6application scopesupports2020Source 1needs review

The FISC system expands the optogenetic toolbox for DNA recombination to enable spatiotemporally controlled, non-invasive genome engineering in living systems.

Thus, the FISC system expands the optogenetic toolbox for DNA recombination to achieve spatiotemporally controlled, non-invasive genome engineering in living systems.
Claim 7application scopesupports2020Source 1needs review

The FISC system expands the optogenetic toolbox for DNA recombination to enable spatiotemporally controlled, non-invasive genome engineering in living systems.

Thus, the FISC system expands the optogenetic toolbox for DNA recombination to achieve spatiotemporally controlled, non-invasive genome engineering in living systems.
Claim 8comparative performancesupports2020Source 1needs review

In vivo, the FISC system shows strong organ penetration and markedly outperforms two blue-light-based Cre systems for recombination induction in the liver.

Our in vivo studies showcase the strong organ-penetration capacity of FISC system, markedly outperforming two blue-light-based Cre systems for recombination induction in the liver.
Claim 9comparative performancesupports2020Source 1needs review

In vivo, the FISC system shows strong organ penetration and markedly outperforms two blue-light-based Cre systems for recombination induction in the liver.

Our in vivo studies showcase the strong organ-penetration capacity of FISC system, markedly outperforming two blue-light-based Cre systems for recombination induction in the liver.
Claim 10comparative performancesupports2020Source 1needs review

In vivo, the FISC system shows strong organ penetration and markedly outperforms two blue-light-based Cre systems for recombination induction in the liver.

Our in vivo studies showcase the strong organ-penetration capacity of FISC system, markedly outperforming two blue-light-based Cre systems for recombination induction in the liver.
Claim 11comparative performancesupports2020Source 1needs review

In vivo, the FISC system shows strong organ penetration and markedly outperforms two blue-light-based Cre systems for recombination induction in the liver.

Our in vivo studies showcase the strong organ-penetration capacity of FISC system, markedly outperforming two blue-light-based Cre systems for recombination induction in the liver.
Claim 12comparative performancesupports2020Source 1needs review

In vivo, the FISC system shows strong organ penetration and markedly outperforms two blue-light-based Cre systems for recombination induction in the liver.

Our in vivo studies showcase the strong organ-penetration capacity of FISC system, markedly outperforming two blue-light-based Cre systems for recombination induction in the liver.
Claim 13comparative performancesupports2020Source 1needs review

In vivo, the FISC system shows strong organ penetration and markedly outperforms two blue-light-based Cre systems for recombination induction in the liver.

Our in vivo studies showcase the strong organ-penetration capacity of FISC system, markedly outperforming two blue-light-based Cre systems for recombination induction in the liver.
Claim 14comparative performancesupports2020Source 1needs review

In vivo, the FISC system shows strong organ penetration and markedly outperforms two blue-light-based Cre systems for recombination induction in the liver.

Our in vivo studies showcase the strong organ-penetration capacity of FISC system, markedly outperforming two blue-light-based Cre systems for recombination induction in the liver.
Claim 15delivery applicationsupports2020Source 1needs review

The FISC system was successfully deployed using adeno-associated virus delivery.

Demonstrating its strong clinical relevance, we successfully deploy a FISC system using adeno-associated virus (AAV) delivery.
Claim 16delivery applicationsupports2020Source 1needs review

The FISC system was successfully deployed using adeno-associated virus delivery.

Demonstrating its strong clinical relevance, we successfully deploy a FISC system using adeno-associated virus (AAV) delivery.
Claim 17delivery applicationsupports2020Source 1needs review

The FISC system was successfully deployed using adeno-associated virus delivery.

Demonstrating its strong clinical relevance, we successfully deploy a FISC system using adeno-associated virus (AAV) delivery.
Claim 18delivery applicationsupports2020Source 1needs review

The FISC system was successfully deployed using adeno-associated virus delivery.

Demonstrating its strong clinical relevance, we successfully deploy a FISC system using adeno-associated virus (AAV) delivery.
Claim 19delivery applicationsupports2020Source 1needs review

The FISC system was successfully deployed using adeno-associated virus delivery.

Demonstrating its strong clinical relevance, we successfully deploy a FISC system using adeno-associated virus (AAV) delivery.
Claim 20delivery applicationsupports2020Source 1needs review

The FISC system was successfully deployed using adeno-associated virus delivery.

Demonstrating its strong clinical relevance, we successfully deploy a FISC system using adeno-associated virus (AAV) delivery.
Claim 21delivery applicationsupports2020Source 1needs review

The FISC system was successfully deployed using adeno-associated virus delivery.

Demonstrating its strong clinical relevance, we successfully deploy a FISC system using adeno-associated virus (AAV) delivery.
Claim 22developmentsupports2020Source 1needs review

The paper reports development of a far-red light-induced split Cre-loxP system called FISC for optogenetic regulation of genome engineering in vivo using far-red light.

Here, we develop a far-red light-induced split Cre-loxP system (FISC system) based on a bacteriophytochrome optogenetic system and split-Cre recombinase, enabling optogenetical regulation of genome engineering in vivo solely by utilizing a far-red light (FRL).
Claim 23developmentsupports2020Source 1needs review

The paper reports development of a far-red light-induced split Cre-loxP system called FISC for optogenetic regulation of genome engineering in vivo using far-red light.

Here, we develop a far-red light-induced split Cre-loxP system (FISC system) based on a bacteriophytochrome optogenetic system and split-Cre recombinase, enabling optogenetical regulation of genome engineering in vivo solely by utilizing a far-red light (FRL).
Claim 24developmentsupports2020Source 1needs review

The paper reports development of a far-red light-induced split Cre-loxP system called FISC for optogenetic regulation of genome engineering in vivo using far-red light.

Here, we develop a far-red light-induced split Cre-loxP system (FISC system) based on a bacteriophytochrome optogenetic system and split-Cre recombinase, enabling optogenetical regulation of genome engineering in vivo solely by utilizing a far-red light (FRL).
Claim 25developmentsupports2020Source 1needs review

The paper reports development of a far-red light-induced split Cre-loxP system called FISC for optogenetic regulation of genome engineering in vivo using far-red light.

Here, we develop a far-red light-induced split Cre-loxP system (FISC system) based on a bacteriophytochrome optogenetic system and split-Cre recombinase, enabling optogenetical regulation of genome engineering in vivo solely by utilizing a far-red light (FRL).
Claim 26developmentsupports2020Source 1needs review

The paper reports development of a far-red light-induced split Cre-loxP system called FISC for optogenetic regulation of genome engineering in vivo using far-red light.

Here, we develop a far-red light-induced split Cre-loxP system (FISC system) based on a bacteriophytochrome optogenetic system and split-Cre recombinase, enabling optogenetical regulation of genome engineering in vivo solely by utilizing a far-red light (FRL).
Claim 27developmentsupports2020Source 1needs review

The paper reports development of a far-red light-induced split Cre-loxP system called FISC for optogenetic regulation of genome engineering in vivo using far-red light.

Here, we develop a far-red light-induced split Cre-loxP system (FISC system) based on a bacteriophytochrome optogenetic system and split-Cre recombinase, enabling optogenetical regulation of genome engineering in vivo solely by utilizing a far-red light (FRL).
Claim 28developmentsupports2020Source 1needs review

The paper reports development of a far-red light-induced split Cre-loxP system called FISC for optogenetic regulation of genome engineering in vivo using far-red light.

Here, we develop a far-red light-induced split Cre-loxP system (FISC system) based on a bacteriophytochrome optogenetic system and split-Cre recombinase, enabling optogenetical regulation of genome engineering in vivo solely by utilizing a far-red light (FRL).
Claim 29performancesupports2020Source 1needs review

The FISC system exhibits low background, no detectable photocytotoxicity, and efficient far-red-light-induced DNA recombination.

The FISC system exhibits low background and no detectable photocytotoxicity, while offering efficient FRL-induced DNA recombination.
Claim 30performancesupports2020Source 1needs review

The FISC system exhibits low background, no detectable photocytotoxicity, and efficient far-red-light-induced DNA recombination.

The FISC system exhibits low background and no detectable photocytotoxicity, while offering efficient FRL-induced DNA recombination.
Claim 31performancesupports2020Source 1needs review

The FISC system exhibits low background, no detectable photocytotoxicity, and efficient far-red-light-induced DNA recombination.

The FISC system exhibits low background and no detectable photocytotoxicity, while offering efficient FRL-induced DNA recombination.
Claim 32performancesupports2020Source 1needs review

The FISC system exhibits low background, no detectable photocytotoxicity, and efficient far-red-light-induced DNA recombination.

The FISC system exhibits low background and no detectable photocytotoxicity, while offering efficient FRL-induced DNA recombination.
Claim 33performancesupports2020Source 1needs review

The FISC system exhibits low background, no detectable photocytotoxicity, and efficient far-red-light-induced DNA recombination.

The FISC system exhibits low background and no detectable photocytotoxicity, while offering efficient FRL-induced DNA recombination.
Claim 34performancesupports2020Source 1needs review

The FISC system exhibits low background, no detectable photocytotoxicity, and efficient far-red-light-induced DNA recombination.

The FISC system exhibits low background and no detectable photocytotoxicity, while offering efficient FRL-induced DNA recombination.
Claim 35performancesupports2020Source 1needs review

The FISC system exhibits low background, no detectable photocytotoxicity, and efficient far-red-light-induced DNA recombination.

The FISC system exhibits low background and no detectable photocytotoxicity, while offering efficient FRL-induced DNA recombination.

Approval Evidence

1 source1 linked approval claimfirst-pass slug adeno-associated-virus-delivery
we successfully deploy a FISC system using adeno-associated virus (AAV) delivery

Source:

delivery applicationsupports

The FISC system was successfully deployed using adeno-associated virus delivery.

Demonstrating its strong clinical relevance, we successfully deploy a FISC system using adeno-associated virus (AAV) delivery.

Source:

Comparisons

Source-backed strengths

The main demonstrated strength is successful in vivo deployment of the FISC system using AAV delivery. Because the associated FISC platform enabled non-invasive genome engineering and showed strong organ penetration with superior liver recombination induction relative to two blue-light-based Cre systems, AAV delivery was compatible with that validated in vivo application.

Ranked Citations

  1. 1.
    StructuralSource 1Nature Communications2020Claim 1Claim 2Claim 3

    Extracted from this source document.