CAG-μDys
Construct Patterntwo engineered AAV9 capsids (N57Q, K51Q) containing CAG-μDys ... Subsequent evaluation of a codon-optimised microdystrophin transgene under the control of the optimal CAG promoter
CFBacMamMusHumTxRep
Ev 26Rep 9Pr 71
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
▾Top-level concepts: biophysical action modes such as heterodimerization, photocleavage, or RNA binding.
Layer 1
Mechanisms
Layer 2
Architectures
▾Arrangements that realize or deploy mechanisms, including switches, construct patterns, and delivery strategies.
Layer 2
Architectures
Layer 3
Components
▾Low-level parts and sequence-defined elements used inside architectures, including protein domains and RNA elements.
Layer 3
Components
Technique Branch
Layer 1
Approaches
▾High-level engineering practices such as computational design, directed evolution, sequence verification, and functional assay.
Layer 1
Approaches
Layer 2
Methods
▾Concrete methods used to design, build, verify, or characterize engineered systems.
Layer 2
Methods
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aav-mediated gene delivery
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sensor cargo
Construct PatternPairing these AAVs with modern gene regulatory elements and state-of-the-art reporter, sensor, and effector cargo enables highly specific transgene expression for anatomical and functional analyses of brain cells and circuits.
CFBacMamMusHumTxRep
Ev 20Rep 9Pr 71
AAV2.NN
Delivery StrategyAAV serotype (AAV2.2 versus engineered AAV2.NN)-were systematically optimized. The engineered AAV2.NN serotype increased transduction efficiency and labeling density under equivalent conditions.
CFBacMamMusHumTxRep
Delandistrogene moxeparvovec
Delivery StrategyDelandistrogene moxeparvovec, the first FDA-approved gene therapy for DMD, has demonstrated transgene expression and potential functional improvement in early phase trials, although its long-term efficacy, durability, and safety remain unconfirmed.
CFBacMamMusHumTxRep
dual AAV
Delivery StrategyRecently, the first-in-human dual AAV therapy for hereditary hearing loss, which overcomes large gene delivery, has showcased the restoration of auditory function for patients.
CFBacMamMusHumTxRep
PHP.eB AAV co-delivery
Delivery StrategyIntravenous injection of two PHP.eB AAVs enabled the whole-brain co-expression of the red-shifted calcium indicator jRCaMP1b and the inhibitory actuator stGtACR2, with stable expression over several weeks.
CFBacMamMusHumTxRep
rapid neonatal AAV delivery
Delivery StrategyHere, we report a fast and efficient technique for delivering adeno-associated virus (AAV) in neonatal pups for the robust co-expression of reporters and sensors in cortical neuronal subpopulations under different promoters and transgenic mouse lines.
CFBacMamMusHumTxRep
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