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UnknownRetinal gene therapy for Stargardt disease with dual AAV intein vectors is both safe and effective in large animal models.
Not available
S-EPMC11939046 | biostudies-literature
UnknownIn Silico Structural Prediction for the Generation of Novel Performant Midi-Dystrophins Based on Intein-Mediated Dual AAV Approach.
Not available
S-EPMC11476470 | biostudies-literature
ProteomicsSplit intein mdx skeletal muscle proteomics
Duchenne muscular dystrophy is a rare and lethal neuromuscular disease caused by loss-of-function mutations in the dystrophin protein that provides structural integrity to striated muscle fibers. Mice with loss-of-function mutations for the Dmd gene encoding dystrophin (mdx-4cv) were treated with mi...
ORGANISM(S): Mus musculus (Mouse) 
2025-11-06 | PXD062324 | Pride
Aav therapy Split-intein Duchenne muscular dystrophy Skeletal muscle Lcms 
Genomics
AAV Delivery of intein-split base editors
PRJNA532891 | ENA
OtherComparison of editing efficiencies of intein, AAVLINK, and StitchR delivery ABE8e systems at the mouse TA
In this study, intein, AAVLINK, and StitchR delivery ABE8e systems were packaged into viral vectors (AAV) and intramuscular injected into mice. Genomic DNA was extracted from the mouse tibialis anterior (TA) muscle, followed by deep sequencing to analyze the editing efficiency.
ORGANISM(S): Mus musculus 
2026-09-30 | GSE348337 | GEO
UnknownInclusion of a degron reduces levelsof undesired inteins after AAV-mediated proteintrans-splicing in the retina.
Not available
S-EPMC8571531 | biostudies-literature
UnknownLiver gene therapy with intein-mediated F8 trans-splicing corrects mouse haemophilia A.
Not available
S-EPMC9174883 | biostudies-literature
UnknownFull-length ATP7B reconstituted through protein trans-splicing corrects Wilson disease in mice.
Not available
S-EPMC9436707 | biostudies-literature
TranscriptomicsRescuing voluntary motor behavior in a Parkinson´s disease model by aCRISPR mediated reprogramming of striatal astrocytes into induced GABAergic neurons
Cell replacement strategies of affected cells have been performed in clinical studies of Parkinson´s disease with variable outcome. Conversion of endogenous glial cells into functional neurons might represent a more reliable alternative, thus here we developed a knock-in mouse line carrying a dual d...
ORGANISM(S): Mus musculus 
2022-03-15 | GSE149872 | GEO
UnknownOptimized dual-AAV base editor delivery system with enhanced editing efficiency and virion production titer.
Not available
S-EPMC12002711 | biostudies-literature
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